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The two documents discuss research into potential new treatments for prion diseases and myotonic dystrophy. The first reports that the protein folding activity of the ribosome (PFAR) plays a key role in prion propagation, and that drugs targeting PFAR show promise as antiprion medicines. The second describes how aberrant splicing in myotonic dystrophy changes a muscle enzyme and fiber types, driving muscle wasting. Both highlight the importance of understanding RNA splicing and protein synthesis defects to develop new therapies for neurodegenerative and muscle diseases.
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SPLICING
The document discusses RNA splicing and its importance. It covers how splicing removes introns and joins exons to create mature RNA for protein production. Alternative splicing can generate multiple protein isoforms from a single gene with different functions. Myotonic dystrophy type 1 is discussed as an example of a disorder caused by aberrant splicing, changing the form of the PKM2 enzyme critical for muscle cell metabolism. A new complex called the Little Elongation Complex is revealed to play a key role in transcription of small nuclear RNAs required for splicing. Understanding splicing machinery could open doors to novel disease treatment approaches.
Rules and impact of nonsense-mediated mRNA decay in human cancers
Premature termination codons (PTCs) cause a large proportion of inherited human genetic diseases. PTC-containing transcripts can be degraded by an mRNA surveillance pathway termed nonsense-mediated mRNA decay (NMD). However, the efficiency of NMD varies; it is inefficient when a PTC is located downstream of the last exon junction complex (EJC). We used matched exome and transcriptome data from 9,769 human tumors to systematically elucidate the rules of NMD targeting in human cells. An integrated model incorporating multiple rules beyond the canonical EJC model explains approximately three-fourths of the non-random variance in NMD efficiency across thousands of PTCs. We also show that dosage compensation may sometimes mask the effects of NMD. Applying the NMD model identifies signatures of both positive and negative selection on NMD-triggering mutations in human tumors and provides a classification for tumor-suppressor genes.
4.28.2010 lecture 2
This document discusses various mechanisms of cancer development and treatment strategies:
1. Cancer cells acquire mutations that allow them to evade normal cell cycle controls and proliferate uncontrollably. Key genes like Ras, Rb, p53 and telomerase are often mutated.
2. Advanced cancer cells recruit blood vessels to fuel their growth, avoid dependence on growth factors, and become invasive. They can also manipulate the immune system to avoid detection.
3. New targeted cancer treatments aim to deliver chemotherapy directly to cancer cells using nanoparticles coated with proteins that bind specifically to receptors on tumor cells. Immunotherapy approaches reprogram a patient's immune cells to attack the cancer.
4. Understanding the hallmarks of
Mutations and Cancer
This document discusses mutations and their relationship to cancer. It begins by defining key terms like mutation, carcinogen, oncogene, and tumor suppressor genes. It then explains how mutations can occur during DNA replication and cell division. Certain mutations in oncogenes and tumor suppressor genes can cause cancer if they result in uncontrolled cell growth and division. The document outlines the typical progression of cancer from a benign tumor to a malignant tumor that can metastasize and spread to other parts of the body. It identifies some known cancer genes and potential oncogenes and tumor suppressor genes. In closing, it notes that cancer development involves both exposure to carcinogens and genetic factors.
Antisense therapy
Antisense therapy uses synthetic nucleic acid strands that bind to messenger RNA (mRNA) to inactivate genes and their protein production. It has potential applications for treating various diseases like cancer, diabetes, and genetic disorders. Antisense oligonucleotides work by binding to mRNA, preventing translation into protein via RNase H activity or steric hindrance. While promising, antisense drugs require further clinical trials to prove efficacy compared to other treatments.
2012 CLN5 Boustany
This study investigated the role of the CLN5 and CLN8 proteins in sphingolipid metabolism and ceramide synthesis. The following key findings are reported:
1) CLN5-deficient fibroblasts showed decreased levels of ceramide, sphingomyelin, and glycosphingolipids as well as defects in adhesion, increased growth, and apoptosis.
2) Expression of CLN8 protein corrected the growth and apoptosis abnormalities in CLN5-deficient cells, suggesting CLN5 and CLN8 proteins interact and modulate ceramide synthase activity.
3) Both CLN5- and CLN8-deficient cells showed decreased de novo ceramide synthesis and specific ceramide
Seminario Biología Molecular
This document summarizes a study on the effects of antimicrobial and anti-cancer therapies on Alzheimer's disease pathogenesis. The study examines how inhibiting alpha-secretase affects p53 status in cultured human neuroblastoma cells. The results suggest targeting both beta-amyloid precursor protein processing and p53 levels could serve as a therapeutic approach by alleviating multiple pathological processes leading to neuronal vulnerability and death. Methodologies included western blot analyses, real-time quantitative PCR, cell viability assays, and flow cytometry. The conclusions determined certain medical therapies could potentially trigger Alzheimer's disease, highlighting the importance of prevention. Studying the consequences of alpha-secretase inhibition provided insights to advance treatment approaches.
Antisense oligonucleotides-therapy-in-the-treatment-of-cerebral-gliomas-a-rev...
This document reviews the use of antisense oligonucleotides for the treatment of cerebral gliomas. It discusses how antisense oligonucleotides work by binding to mRNA and blocking protein synthesis. Genes involved in gliomagenesis like growth factors and their receptors are potential targets. While current glioma treatments have limitations, antisense oligonucleotides may provide a targeted therapy by selectively blocking genes involved in tumor growth and progression. The review examines both clinical and experimental studies on antisense oligonucleotide therapy for cerebral gliomas.
Recommended
SPLICING
The document discusses RNA splicing and its importance. It covers how splicing removes introns and joins exons to create mature RNA for protein production. Alternative splicing can generate multiple protein isoforms from a single gene with different functions. Myotonic dystrophy type 1 is discussed as an example of a disorder caused by aberrant splicing, changing the form of the PKM2 enzyme critical for muscle cell metabolism. A new complex called the Little Elongation Complex is revealed to play a key role in transcription of small nuclear RNAs required for splicing. Understanding splicing machinery could open doors to novel disease treatment approaches.
Rules and impact of nonsense-mediated mRNA decay in human cancers
Premature termination codons (PTCs) cause a large proportion of inherited human genetic diseases. PTC-containing transcripts can be degraded by an mRNA surveillance pathway termed nonsense-mediated mRNA decay (NMD). However, the efficiency of NMD varies; it is inefficient when a PTC is located downstream of the last exon junction complex (EJC). We used matched exome and transcriptome data from 9,769 human tumors to systematically elucidate the rules of NMD targeting in human cells. An integrated model incorporating multiple rules beyond the canonical EJC model explains approximately three-fourths of the non-random variance in NMD efficiency across thousands of PTCs. We also show that dosage compensation may sometimes mask the effects of NMD. Applying the NMD model identifies signatures of both positive and negative selection on NMD-triggering mutations in human tumors and provides a classification for tumor-suppressor genes.
4.28.2010 lecture 2
This document discusses various mechanisms of cancer development and treatment strategies:
1. Cancer cells acquire mutations that allow them to evade normal cell cycle controls and proliferate uncontrollably. Key genes like Ras, Rb, p53 and telomerase are often mutated.
2. Advanced cancer cells recruit blood vessels to fuel their growth, avoid dependence on growth factors, and become invasive. They can also manipulate the immune system to avoid detection.
3. New targeted cancer treatments aim to deliver chemotherapy directly to cancer cells using nanoparticles coated with proteins that bind specifically to receptors on tumor cells. Immunotherapy approaches reprogram a patient's immune cells to attack the cancer.
4. Understanding the hallmarks of
Mutations and Cancer
This document discusses mutations and their relationship to cancer. It begins by defining key terms like mutation, carcinogen, oncogene, and tumor suppressor genes. It then explains how mutations can occur during DNA replication and cell division. Certain mutations in oncogenes and tumor suppressor genes can cause cancer if they result in uncontrolled cell growth and division. The document outlines the typical progression of cancer from a benign tumor to a malignant tumor that can metastasize and spread to other parts of the body. It identifies some known cancer genes and potential oncogenes and tumor suppressor genes. In closing, it notes that cancer development involves both exposure to carcinogens and genetic factors.
Antisense therapy
Antisense therapy uses synthetic nucleic acid strands that bind to messenger RNA (mRNA) to inactivate genes and their protein production. It has potential applications for treating various diseases like cancer, diabetes, and genetic disorders. Antisense oligonucleotides work by binding to mRNA, preventing translation into protein via RNase H activity or steric hindrance. While promising, antisense drugs require further clinical trials to prove efficacy compared to other treatments.
2012 CLN5 Boustany
This study investigated the role of the CLN5 and CLN8 proteins in sphingolipid metabolism and ceramide synthesis. The following key findings are reported:
1) CLN5-deficient fibroblasts showed decreased levels of ceramide, sphingomyelin, and glycosphingolipids as well as defects in adhesion, increased growth, and apoptosis.
2) Expression of CLN8 protein corrected the growth and apoptosis abnormalities in CLN5-deficient cells, suggesting CLN5 and CLN8 proteins interact and modulate ceramide synthase activity.
3) Both CLN5- and CLN8-deficient cells showed decreased de novo ceramide synthesis and specific ceramide
Seminario Biología Molecular
This document summarizes a study on the effects of antimicrobial and anti-cancer therapies on Alzheimer's disease pathogenesis. The study examines how inhibiting alpha-secretase affects p53 status in cultured human neuroblastoma cells. The results suggest targeting both beta-amyloid precursor protein processing and p53 levels could serve as a therapeutic approach by alleviating multiple pathological processes leading to neuronal vulnerability and death. Methodologies included western blot analyses, real-time quantitative PCR, cell viability assays, and flow cytometry. The conclusions determined certain medical therapies could potentially trigger Alzheimer's disease, highlighting the importance of prevention. Studying the consequences of alpha-secretase inhibition provided insights to advance treatment approaches.
Antisense oligonucleotides-therapy-in-the-treatment-of-cerebral-gliomas-a-rev...
This document reviews the use of antisense oligonucleotides for the treatment of cerebral gliomas. It discusses how antisense oligonucleotides work by binding to mRNA and blocking protein synthesis. Genes involved in gliomagenesis like growth factors and their receptors are potential targets. While current glioma treatments have limitations, antisense oligonucleotides may provide a targeted therapy by selectively blocking genes involved in tumor growth and progression. The review examines both clinical and experimental studies on antisense oligonucleotide therapy for cerebral gliomas.
The-Genetic-code-converted.pptx
The genetic code is the set of rules by which genetic material like DNA and mRNA are translated into amino acid sequences that make up proteins. The genetic code specifies which amino acid will be added next during protein synthesis using sequences of three nucleotides called codons. While some codons specify the same amino acid, meaning the code is degenerate, this allows more codons to exist without increasing the number of amino acids used. Efforts to understand the genetic code increased after the discovery of DNA's structure, with scientists like Gamow theorizing it must use three nucleotides to code for the 20 standard amino acids.
Jocelyn abstract conference Jan 2016
Three mouse lines were generated using TALENs to model the bovine Developmental Duplications condition in cattle. Mice with mutations in the orthologous NHLRC2 gene showed embryonic lethality when homozygous. Heterozygous mice were mated and genotyping of offspring revealed an absence of homozygous individuals, suggesting embryonic death before day E8.5 of development. Analysis of embryos at earlier developmental stages is ongoing to determine the stage at which lethality occurs.
CRISPR cas9 mediated TERT disruption in cancer cells
CRISPR/Cas9 was used to disrupt the TERT gene in cancer cells. Three gRNAs targeted exon regions of the TERT gene in HELA, PANC1, and SKBR3 cells, achieving up to 85% editing efficiency as determined by PCR and T7 endonuclease assays. Xenograft experiments found that editing TERT prevented tumor formation in nude mice. While effective, delivery methods need improvement and off-target effects require validation before clinical use. Future work aims to identify additional tumor-specific genes that could be targeted with CRISPR to develop new cancer therapies.
Antisense therapy
Antisense therapy is a form of genetic treatment that uses artificially synthesized nucleic acid sequences to bind to messenger RNA (mRNA) produced by a disease-causing gene, inactivating it and effectively turning the gene "off". This approach is being researched as a treatment for various diseases including cancers, diabetes, ALS, and muscular dystrophy. Antisense drugs work by forming DNA/RNA hybrids that are degraded by the RNase H enzyme, preventing translation of the mRNA into protein.
Somatic activating kras mutations in arteriovenous malformation
The document discusses a study investigating somatic activating KRAS mutations in arteriovenous malformations. The study aimed to determine if KRAS mutation is a genetic marker for arteriovenous malformation and analyze the relationship between abnormal MAPK-EPK signaling and tissue defects. The study analyzed 72 patients and found somatic activating KRAS mutations in arteriovenous malformation tissue samples. The findings suggest KRAS mutation may be a genetic factor associated with arteriovenous malformations.
2014 BDSRA Storch CLN7
1. The document summarizes research on CLN7 disease, which is caused by mutations in the CLN7 gene encoding a lysosomal membrane protein.
2. A mouse model of CLN7 disease was generated that recapitulates some neuropathological features seen in human patients, including accumulation of autofluorescent storage material in the brain.
3. The researchers aim to identify the pathomechanisms of CLN7 disease, develop biomarkers, analyze the function of the CLN7 protein, and understand how mutations impact its localization and function to inform potential therapies.
ANTISENSE OLIGONUCLEOTIDES
This document discusses antisense oligonucleotides, which are single-stranded RNA molecules that regulate gene expression by binding to mRNA. Antisense oligonucleotides have been developed as therapeutics to treat conditions like cytomegalovirus retinitis, hypercholesterolemia, and muscular dystrophy. They work either by recruiting RNase H to degrade mRNA or by physically blocking translation. Delivery of antisense oligonucleotides into cells occurs through endocytosis or fluid-phase pinocytosis. Antisense technologies can be used in drug discovery to specifically manipulate gene expression and identify novel drug targets.
MicroarrayAnalysisinR_2014
This study generated a custom neuro-ncRNA microarray to profile non-coding RNA expression in mouse models of neurological diseases. They identified ncRNAs from RNA-Seq data and computational prediction, and validated tissue-specific expression. Using the microarray, they found differential ncRNA expression in mouse models of psychiatric disorders and Parkinson's disease with impaired calcium channel activity, as well as in an Alzheimer's disease mouse model. The neuro-ncRNA microarray provides a sensitive tool for efficiently analyzing ncRNA expression changes related to central nervous system diseases.
Microbiology.
Prions are proteinaceous pathogens that cause transmissible spongiform encephalopathies (TSEs) by changing their structure from a normal alpha helix form (PrPC) to an abnormal beta-pleated sheet form (PrPSc). Current treatments aim to reduce PrPC conversion through RNA interference or active DNA vaccination, but these have limitations. While RNAi risks saturating proteins and reducing long-term effectiveness, active DNA vaccination may delay but not prevent disease progression. Chemically modified oligonucleotides can also bind PrPC and prevent conversion, but their effects are very toxic. The best current option is active DNA vaccination, as it delays onset without adverse side effects like the other treatments.
Seminario Laura Agudelo Vergara
This document summarizes research investigating the role of SREBP-2 in breast cancer metastasis to bone. SREBP-2 was found to positively regulate osteoclast formation and bone resorption in vitro. It also regulates the expression of NFATc1 during osteoclastogenesis through the RANKL/CREB signaling pathway. As SREBP-2 promotes factors involved in bone destruction and breast cancer spread, inhibiting SREBP-2 may be a potential therapeutic approach for breast cancer patients with bone metastases.
Single nucleotide polymorphism (sn ps)
Single nucleotide polymorphisms (SNPs) are variations in single nucleotides that occur at specific positions in the genome. For example, the base C may appear in most individuals at a specific position, but in some individuals the position is occupied by base A, representing a SNP. SNPs can be classified as synonymous or nonsynonymous depending on whether they change the amino acid sequence. Common human diseases like sickle cell anemia and cystic fibrosis result from SNPs. SNPs can be identified and analyzed using methods like DNA sequencing, mass spectrometry, and primer extension.
THE GUARDIAN ANGEL OF GENOME P53 IN CANCER TREATMENT
Currently, around 11 million people are living with a tumour that contains an inactivating mutation of TP53 (the human gene that encodes p53) and another 11 million have tumours in which the p53 pathway is partially abrogated through the inactivation of other signalling or effector components. The p53 pathway is therefore a prime target for new cancer drug development, and several original approaches to drug discovery that could have wide applications to drug development are being used. In one approach, molecules that activate p53 by blocking protein–protein interactions with MDM2 are in early clinical development. Remarkable progress has also been made in the development of p53-binding molecules that can rescue the function of certain p53 mutants.
Tools used in molecular biology
This document provides information about genetic polymorphisms and methods used to detect single nucleotide polymorphisms (SNPs), including DNA isolation, polymerase chain reaction (PCR), restriction fragment length polymorphism (RFLP), and gel electrophoresis. It defines a polymorphism as a harmless DNA variation between individuals and SNPs as single base mutations that are the most common source of genetic variation. The document outlines the steps of PCR including melting, annealing, and extension cycles to amplify DNA, as well as how restriction enzymes are used to digest PCR products into different fragments depending on sequences, which can then be separated by size via gel electrophoresis to detect SNPs.
Antisense oligoneucleotides in therapy
This document discusses antisense oligonucleotides, which are synthetic genetic molecules that bind to mRNA to prevent protein translation. They work by either blocking access of the translation machinery or inducing RNase H to degrade the mRNA. The document outlines their mechanism of action, advantages like rapid design and potential for enhanced binding, and challenges like delivery and toxicity. It provides examples of antisense oligonucleotide therapies for conditions like cancer, asthma, Duchenne muscular dystrophy, diabetes, hemorrhagic fever viruses, HIV/AIDS, and cytomegalovirus retinitis.
Cancer-an overview by Prof Viyatprajna Acharya, KIMS, Bhubaneswar
This presentation is targeted for MBBS, MD and BDS students that describes briefly about aetiopathogenesis, tumour markers, anti cancer agents, apoptosis
Antisense rna and dna
This document discusses antisense RNA and DNA technology. It explains that antisense works by introducing short DNA or RNA sequences that are complementary to target mRNA, preventing translation into protein. This can inhibit genetic disorders caused by mutated proteins. The document provides examples of using antisense to treat diseases like cancer and viruses. It describes various methods of delivering antisense sequences into cells and notes that while promising, most antisense therapies have yet to produce significant clinical results, though one was approved by the FDA to treat cytomegalovirus retinitis.
Markus Zweckstetter
Presentation made by Dr. Markus Zweckstetter on October 30, 2015 at the Alzforum-hosted live webinar titled "Fluid Business: Could “Liquid” Protein Herald Neurodegeneration?"
More information and the recording of the session available at http://www.alzforum.org/webinars/fluid-business-could-liquid-protein-herald-neurodegeneration
Antisense oligonucleotide therapy
This document provides an overview of antisense oligonucleotide therapy, including its definition as synthetic genetic material that binds to mRNA to prevent protein translation, mechanisms of action such as blocking ribosomes and activating RNase enzymes, applications in oncology and other therapeutic areas, examples of compounds in clinical trials, and its future potential as over 30 compounds are currently in clinical trials.
Antisense technologies and antisense oligonucleotides
Antisense oligonucleotides are short strands of nucleic acids that bind to messenger RNA (mRNA) and inhibit gene expression. There are three main approaches for antisense oligonucleotides based on their ability to activate RNase H enzyme and resist nucleases. Antisense oligonucleotides show potential for treating various diseases like cancer, neurological disorders, and viral infections by hybridizing to target mRNA. However, challenges remain around protecting the oligonucleotides from degradation and improving cellular uptake and target specificity. Overall, antisense technology provides a novel approach for modulating gene expression and developing new therapies.
Anti sense drugs
Antisense drugs work by binding to mRNA to prevent protein production from mutated genes causing genetic disorders. They are short DNA sequences that match the mRNA sequence. First generation antisense oligonucleotides used phosphorothioate modifications but had toxicity issues. Second generation ones use 2'-O-methyl and 2'-O-methoxyethyl modifications, showing better stability and less toxicity. Newer third generation modifications like PNA and LNA further increase binding affinity and stability. Effective delivery remains a challenge, with liposomes and nanoparticles being investigated to protect antisense drugs and deliver them to target tissues.
Post tranlational modification
Posttranslational modifications occur in different locations in the cell and serve various purposes. The main locations for modification include the nucleus, lysosome, mitochondria, Golgi, ER, cytosol, ribosome, plasma membrane, extracellular fluid, and extracellular matrix. Common types of modifications include acetylation, phosphorylation, glycosylation, amidation, and ubiquitination. These modifications influence protein stability, activity, localization, and signaling interactions.
Research report (alternative splicing, protein structure; retinitis pigmentosa)
This presentation explains the two major scientific projects I have been involved in.
It extends way further than a CV, but shorter than an actual scientific paper.
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The-Genetic-code-converted.pptx
The genetic code is the set of rules by which genetic material like DNA and mRNA are translated into amino acid sequences that make up proteins. The genetic code specifies which amino acid will be added next during protein synthesis using sequences of three nucleotides called codons. While some codons specify the same amino acid, meaning the code is degenerate, this allows more codons to exist without increasing the number of amino acids used. Efforts to understand the genetic code increased after the discovery of DNA's structure, with scientists like Gamow theorizing it must use three nucleotides to code for the 20 standard amino acids.
Jocelyn abstract conference Jan 2016
Three mouse lines were generated using TALENs to model the bovine Developmental Duplications condition in cattle. Mice with mutations in the orthologous NHLRC2 gene showed embryonic lethality when homozygous. Heterozygous mice were mated and genotyping of offspring revealed an absence of homozygous individuals, suggesting embryonic death before day E8.5 of development. Analysis of embryos at earlier developmental stages is ongoing to determine the stage at which lethality occurs.
CRISPR cas9 mediated TERT disruption in cancer cells
CRISPR/Cas9 was used to disrupt the TERT gene in cancer cells. Three gRNAs targeted exon regions of the TERT gene in HELA, PANC1, and SKBR3 cells, achieving up to 85% editing efficiency as determined by PCR and T7 endonuclease assays. Xenograft experiments found that editing TERT prevented tumor formation in nude mice. While effective, delivery methods need improvement and off-target effects require validation before clinical use. Future work aims to identify additional tumor-specific genes that could be targeted with CRISPR to develop new cancer therapies.
Antisense therapy
Antisense therapy is a form of genetic treatment that uses artificially synthesized nucleic acid sequences to bind to messenger RNA (mRNA) produced by a disease-causing gene, inactivating it and effectively turning the gene "off". This approach is being researched as a treatment for various diseases including cancers, diabetes, ALS, and muscular dystrophy. Antisense drugs work by forming DNA/RNA hybrids that are degraded by the RNase H enzyme, preventing translation of the mRNA into protein.
Somatic activating kras mutations in arteriovenous malformation
The document discusses a study investigating somatic activating KRAS mutations in arteriovenous malformations. The study aimed to determine if KRAS mutation is a genetic marker for arteriovenous malformation and analyze the relationship between abnormal MAPK-EPK signaling and tissue defects. The study analyzed 72 patients and found somatic activating KRAS mutations in arteriovenous malformation tissue samples. The findings suggest KRAS mutation may be a genetic factor associated with arteriovenous malformations.
2014 BDSRA Storch CLN7
1. The document summarizes research on CLN7 disease, which is caused by mutations in the CLN7 gene encoding a lysosomal membrane protein.
2. A mouse model of CLN7 disease was generated that recapitulates some neuropathological features seen in human patients, including accumulation of autofluorescent storage material in the brain.
3. The researchers aim to identify the pathomechanisms of CLN7 disease, develop biomarkers, analyze the function of the CLN7 protein, and understand how mutations impact its localization and function to inform potential therapies.
ANTISENSE OLIGONUCLEOTIDES
This document discusses antisense oligonucleotides, which are single-stranded RNA molecules that regulate gene expression by binding to mRNA. Antisense oligonucleotides have been developed as therapeutics to treat conditions like cytomegalovirus retinitis, hypercholesterolemia, and muscular dystrophy. They work either by recruiting RNase H to degrade mRNA or by physically blocking translation. Delivery of antisense oligonucleotides into cells occurs through endocytosis or fluid-phase pinocytosis. Antisense technologies can be used in drug discovery to specifically manipulate gene expression and identify novel drug targets.
MicroarrayAnalysisinR_2014
This study generated a custom neuro-ncRNA microarray to profile non-coding RNA expression in mouse models of neurological diseases. They identified ncRNAs from RNA-Seq data and computational prediction, and validated tissue-specific expression. Using the microarray, they found differential ncRNA expression in mouse models of psychiatric disorders and Parkinson's disease with impaired calcium channel activity, as well as in an Alzheimer's disease mouse model. The neuro-ncRNA microarray provides a sensitive tool for efficiently analyzing ncRNA expression changes related to central nervous system diseases.
Microbiology.
Prions are proteinaceous pathogens that cause transmissible spongiform encephalopathies (TSEs) by changing their structure from a normal alpha helix form (PrPC) to an abnormal beta-pleated sheet form (PrPSc). Current treatments aim to reduce PrPC conversion through RNA interference or active DNA vaccination, but these have limitations. While RNAi risks saturating proteins and reducing long-term effectiveness, active DNA vaccination may delay but not prevent disease progression. Chemically modified oligonucleotides can also bind PrPC and prevent conversion, but their effects are very toxic. The best current option is active DNA vaccination, as it delays onset without adverse side effects like the other treatments.
Seminario Laura Agudelo Vergara
This document summarizes research investigating the role of SREBP-2 in breast cancer metastasis to bone. SREBP-2 was found to positively regulate osteoclast formation and bone resorption in vitro. It also regulates the expression of NFATc1 during osteoclastogenesis through the RANKL/CREB signaling pathway. As SREBP-2 promotes factors involved in bone destruction and breast cancer spread, inhibiting SREBP-2 may be a potential therapeutic approach for breast cancer patients with bone metastases.
Single nucleotide polymorphism (sn ps)
Single nucleotide polymorphisms (SNPs) are variations in single nucleotides that occur at specific positions in the genome. For example, the base C may appear in most individuals at a specific position, but in some individuals the position is occupied by base A, representing a SNP. SNPs can be classified as synonymous or nonsynonymous depending on whether they change the amino acid sequence. Common human diseases like sickle cell anemia and cystic fibrosis result from SNPs. SNPs can be identified and analyzed using methods like DNA sequencing, mass spectrometry, and primer extension.
THE GUARDIAN ANGEL OF GENOME P53 IN CANCER TREATMENT
Currently, around 11 million people are living with a tumour that contains an inactivating mutation of TP53 (the human gene that encodes p53) and another 11 million have tumours in which the p53 pathway is partially abrogated through the inactivation of other signalling or effector components. The p53 pathway is therefore a prime target for new cancer drug development, and several original approaches to drug discovery that could have wide applications to drug development are being used. In one approach, molecules that activate p53 by blocking protein–protein interactions with MDM2 are in early clinical development. Remarkable progress has also been made in the development of p53-binding molecules that can rescue the function of certain p53 mutants.
Tools used in molecular biology
This document provides information about genetic polymorphisms and methods used to detect single nucleotide polymorphisms (SNPs), including DNA isolation, polymerase chain reaction (PCR), restriction fragment length polymorphism (RFLP), and gel electrophoresis. It defines a polymorphism as a harmless DNA variation between individuals and SNPs as single base mutations that are the most common source of genetic variation. The document outlines the steps of PCR including melting, annealing, and extension cycles to amplify DNA, as well as how restriction enzymes are used to digest PCR products into different fragments depending on sequences, which can then be separated by size via gel electrophoresis to detect SNPs.
Antisense oligoneucleotides in therapy
This document discusses antisense oligonucleotides, which are synthetic genetic molecules that bind to mRNA to prevent protein translation. They work by either blocking access of the translation machinery or inducing RNase H to degrade the mRNA. The document outlines their mechanism of action, advantages like rapid design and potential for enhanced binding, and challenges like delivery and toxicity. It provides examples of antisense oligonucleotide therapies for conditions like cancer, asthma, Duchenne muscular dystrophy, diabetes, hemorrhagic fever viruses, HIV/AIDS, and cytomegalovirus retinitis.
Cancer-an overview by Prof Viyatprajna Acharya, KIMS, Bhubaneswar
This presentation is targeted for MBBS, MD and BDS students that describes briefly about aetiopathogenesis, tumour markers, anti cancer agents, apoptosis
Antisense rna and dna
This document discusses antisense RNA and DNA technology. It explains that antisense works by introducing short DNA or RNA sequences that are complementary to target mRNA, preventing translation into protein. This can inhibit genetic disorders caused by mutated proteins. The document provides examples of using antisense to treat diseases like cancer and viruses. It describes various methods of delivering antisense sequences into cells and notes that while promising, most antisense therapies have yet to produce significant clinical results, though one was approved by the FDA to treat cytomegalovirus retinitis.
Markus Zweckstetter
Presentation made by Dr. Markus Zweckstetter on October 30, 2015 at the Alzforum-hosted live webinar titled "Fluid Business: Could “Liquid” Protein Herald Neurodegeneration?"
More information and the recording of the session available at http://www.alzforum.org/webinars/fluid-business-could-liquid-protein-herald-neurodegeneration
Antisense oligonucleotide therapy
This document provides an overview of antisense oligonucleotide therapy, including its definition as synthetic genetic material that binds to mRNA to prevent protein translation, mechanisms of action such as blocking ribosomes and activating RNase enzymes, applications in oncology and other therapeutic areas, examples of compounds in clinical trials, and its future potential as over 30 compounds are currently in clinical trials.
Antisense technologies and antisense oligonucleotides
Antisense oligonucleotides are short strands of nucleic acids that bind to messenger RNA (mRNA) and inhibit gene expression. There are three main approaches for antisense oligonucleotides based on their ability to activate RNase H enzyme and resist nucleases. Antisense oligonucleotides show potential for treating various diseases like cancer, neurological disorders, and viral infections by hybridizing to target mRNA. However, challenges remain around protecting the oligonucleotides from degradation and improving cellular uptake and target specificity. Overall, antisense technology provides a novel approach for modulating gene expression and developing new therapies.
Anti sense drugs
Antisense drugs work by binding to mRNA to prevent protein production from mutated genes causing genetic disorders. They are short DNA sequences that match the mRNA sequence. First generation antisense oligonucleotides used phosphorothioate modifications but had toxicity issues. Second generation ones use 2'-O-methyl and 2'-O-methoxyethyl modifications, showing better stability and less toxicity. Newer third generation modifications like PNA and LNA further increase binding affinity and stability. Effective delivery remains a challenge, with liposomes and nanoparticles being investigated to protect antisense drugs and deliver them to target tissues.
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Cancer-an overview by Prof Viyatprajna Acharya, KIMS, Bhubaneswar
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Post tranlational modification
Posttranslational modifications occur in different locations in the cell and serve various purposes. The main locations for modification include the nucleus, lysosome, mitochondria, Golgi, ER, cytosol, ribosome, plasma membrane, extracellular fluid, and extracellular matrix. Common types of modifications include acetylation, phosphorylation, glycosylation, amidation, and ubiquitination. These modifications influence protein stability, activity, localization, and signaling interactions.
Research report (alternative splicing, protein structure; retinitis pigmentosa)
This presentation explains the two major scientific projects I have been involved in.
It extends way further than a CV, but shorter than an actual scientific paper.
340 - 05 -- processing
This document discusses various topics related to gene expression and regulation, including alternative mRNA splicing, RNA processing, and epigenetic mechanisms like DNA methylation. It explains that primary mRNA transcripts contain both exons and introns, and the process of splicing removes the introns and joins the exons to produce a final mRNA that can be translated into protein. Alternative splicing allows single genes to code for multiple proteins. The document also discusses other post-transcriptional modifications like capping and polyadenylation, as well as epigenetic factors and genetic imprinting.
Dna Profiling Using Pcr Notes
The document discusses DNA profiling using PCR. It explains that DNA contains non-coding regions with repetitive sequences called microsatellites or STRs that vary in length between individuals. PCR is used to target and amplify multiple polymorphic STR sites, which allows for unique DNA profiles to be generated even when considering that individuals may share alleles at some loci. Comparing the lengths of STRs amplified from a sample to a reference can determine if two DNA sources match or not.
Introns: structure and functions
"Introns: Structure and Functions" during November, 2011 (Friday Seminar activity, Department of Biotechnology, University of Agricultural Sciences, Dharwad, Karnataka) by Yogesh S Bhagat (Ph D Scholar)
Genome origin
The document discusses several topics related to genome evolution, including:
1. Genome evolution involves the acquisition of new genes through gene duplication within genomes or lateral gene transfer between species. Gene duplication can occur at the whole genome level or for individual or groups of genes.
2. Genome evolution also involves rearrangement of existing genes through domain shuffling, where gene segments are joined to form new proteins, or domain duplication where repetitive domains evolve new functions.
3. Introns may have evolved early in ancestral genes and are gradually being lost from genomes ("introns early" hypothesis) or evolved more recently and are accumulating ("introns late"). Early evidence supported "introns early" with conserved intron positions in homologous genes.
Cytogenetics 1
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Genome organisation in eukaryotes...........!!!!!!!!!!!
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Genome organisation in eukaryotes...........!!!!!!!!!!!
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Plegable
- 1. SARA OCAMPO RAMÍREZ MEDICINE STUDENT
- 4. mRNA SPLICING Exons Introns mRNA ready for protein synthesis Protein synthesis mRNA tRNA RIBOSOME Proteins -Mad cow -Creutzdeldt-Jackob Muscular dystrophy
- 5. The Ribosome: New Target for Antiprion Medicines July 2, 2103 The inadequate knowledge in the field about the factors involved in prion formation makes the discovery of effective medicines for prion diseases rather challenging.
- 6. The PFAR (protein folding activity of the ribosome) plays a great role in the prions propagation, who do that it’s considerate how good blank for antiprion, in the those diseases treatment. Damages in the prion can generate neurodegenerative diseases in human, like mad cow and Creutzfeldt-Jakob
- 7. In the ribosome happen the protein synthesis of the cell. PFAR is a rRNA dependent of the large subunit of the ribosome. The PFAR center closely overlaps the peptidyl transferase center although the nucleobases responsible for these two functions are not all common.
- 8. The 6-aminophenanthridine and guanabenz acetate implement antiprion activity by binding to ribosomal RNA and inhibiting PFAR. Target for antiprion medicines
- 9. In my opinion those studies about prion can heal neurodegenerative diseases. Is very interesting know new treatments for try on prion, just with PFAR activity. Other advantage, is its medicament price and its action, what can assure better results. and a better life quality.
- 10. Aberrant Splicing Saps the Strength of 'Slow' Muscle Fibers July 29, 2103 Muscle fibers Fast Slow or type 1 In people with myotonic dystrophy these fibers don’t work well
- 11. In the muscle, in normal conditions, the PKM (enzyme who participate in cell muscle metabolism), in patients with myotonic dystrophy reverts to the embryonic form (PKM2), which changes its activity in the cell. This enzyme represents a shift back to the fetal splicing pattern," Cooper.
- 12. About the studies… "We don't know what it is doing to the metabolism, but it seems to be pushing it in the opposite direction from what slow fibers do," said Cooper. "This is related to the loss of slow fibers in myotonic dystrophy.“ Not always the alternative splicing is cancer.
- 13. Myotonic dystrophy occurs when the nucleotides CTG (cytosine, thymine, guanine) repeat an abnormal number of times. When the CTG in the DNA is transcribed into CUG in RNA, the resulting aberrant protein is toxic and disrupts the activity of RNA factors (MBNL1 AND CELF1), which are two RNA splicing factors. The resultant splicing changes somehow drive the skeletal and heart muscle wasting seen in the disease. CTG DNA Transcribed CUG RNA Wrong protein MBNL1 CELF1 DNA
- 14. In my opinion the discover a new illness caused by alternative splicing different to cancer, open ideas in scientific studies, letting understand other type of illness like myotonic dystrophy diseases and implement new treatments in a future. Is important recognize the metabolism role in all this alternative splicing.
- 16. Both studies contributed interesting problems caused by damages in RNA splicing or proteins synthesis, and how the not correct process can unchain different diseases.
- 17. The fact to try to understand that, let make new treatments in patients with one of those alterations.
- 18. The really interesting thing is that the treatment, in the case of myotonic dystrophy, is not very expensive and provide better quality of life in these patients.
- 19. • Journal reference: Meg Byron, Phillip D. Gregory, Jun Jiang, Allison Cotton, Carolyn Brown, David Shivack, Lisa Hall. www.sciencesdirect.com. News Available in: http://www.sciencedaily.com/releases/2013/07/130702100120.htm • Journal reference: K. Linhart, H. D. Novoleva, Carl Sharon, Nick Street, Gregory Hall, Sarah Catn. www.sciencesdirect.com. News Available in: http://www.sciencedaily.com/releases/2013/07/130729161755.htm • MARTINEZ SÁNCHEZ, Lina María. Biología molecular. 2. ed. Medellín: UPB. Fac. de Medicina, 2006. 208 p.