Genetic screening and gene therapy can detect and treat genetic disorders. Genetic screening techniques like carrier screening, newborn screening, and prenatal diagnosis identify genetic risks. Gene therapy aims to treat diseases by replacing faulty genes, such as inserting functional genes into somatic cells. While promising, gene therapy faces challenges like safety concerns over viral vectors and ensuring proper expression of inserted genes. Genetic counseling educates patients on genetic risks and testing options.
"Epigenetics refers to genetic factors that change an organism’s appearance or biological functions without changing the actual DNA sequence. In other words, gene expression changes but the genes themselves don’t. Epigenetics adds an additional level of complexity to the genetic code." - Public Health Cafe
HGP was conceived in 1984 & officially begun in earnest in October 1990.
HGP is a large multicentric, international collaborative venture, the main aim of which is to determine the nucleotide sequence of the entire human nuclear genome.
In 1997, United States established the National Human Genome Research Institute (NHGRI).
The HGP was an international research groups from six countries- USA, UK, France, Germany, Japan and China, & several laboratories and a large no. of scientists and technicians from various disciplines.
Epigenetics is the study, in the field of genetics, of cellular and physiological phenotypic trait variations that are caused by external or environmental factors that switch genes on and off and affect how cells read genes instead of being caused by changes in the DNA sequence. -Wikipedia
"Epigenetics refers to genetic factors that change an organism’s appearance or biological functions without changing the actual DNA sequence. In other words, gene expression changes but the genes themselves don’t. Epigenetics adds an additional level of complexity to the genetic code." - Public Health Cafe
HGP was conceived in 1984 & officially begun in earnest in October 1990.
HGP is a large multicentric, international collaborative venture, the main aim of which is to determine the nucleotide sequence of the entire human nuclear genome.
In 1997, United States established the National Human Genome Research Institute (NHGRI).
The HGP was an international research groups from six countries- USA, UK, France, Germany, Japan and China, & several laboratories and a large no. of scientists and technicians from various disciplines.
Epigenetics is the study, in the field of genetics, of cellular and physiological phenotypic trait variations that are caused by external or environmental factors that switch genes on and off and affect how cells read genes instead of being caused by changes in the DNA sequence. -Wikipedia
THIS PRESENATATION IS FOR THE MEDICAL STUDENTS WHO ALSO HAVE GENETICS AND IF THEY NEED TO GIVE A SEMINAR BASED ON THIS TOPIC THIS PRESENATATION SHALL PROVE USEFUL
Molecular diagnosis of genetic disease ppt for studentsthirupathiSathya
DEFINITION:
Dna analysis can be used for the identification of carriers of hereditary disorders.
For prenatal diagnosis of serious genetic conditions yearly diagnosis before the onset of symptoms is done MOLECULAR DIAGNOSIS OF GENETIC DISEASE
CYSTIC FIBROSIS:
Cystic fibrosis is a genetic disease that affect mostly lungs and also the pancreas.
Screening test:
It is a complex process
Large number of genetic alterations have to be done.
For eg : It is the one of the most common lethal autosomal recessive disorder in Europe.
It is caused by mutations to cystic fibrosis transmembrane conductance regulator(CFTR) gene .
Screening individuals who may be at risk for cystic fibrosis for 500 different mutations is a daunting task.
Diagnosis test that screen for a large number of mutations of a single gene in one assay being developed.
SICKLE CELL ANEMIA:
It is a disorder where red blood cells become rigid and sticky and are shaped like “sickle”.
This irregularly shaped cells stucks in small blood vessels which can slow and block the blood flow and oxygen to all the parts of the body.
There’s no cure for sickle cell anemia.
Screening for sickle cell anemia:
SCA is a genetic disease that is the result of a single nucleotide change in the codon for the sixth aminoacid of the β- chain of the hemoglobin molecule.
The anemia is caused by the inability of the mutated hemoglobin to carry sufficient oxygen.
Target – probe hybridasation is done.
This presentation on Epigenetics is most advanced and evidence based one. Its Very helpful for Genetics students and research fellows, Reproductive Medicine specialist, Reproductive Biologist, Infertility practitioners
Autosomal recessive inheritance refers to the pattern of inheritance of a condition directly or indirectly due to a recessive faulty gene copy located on an autosome Conditions that follow a pattern of autosomal recessive inheritance usually affect men and women equally and include cystic fibrosis, thalassaemia, Tay-Sachs disease and haemochromatosis. These autosomal recessive conditions are more common in individuals of certain ethnic or cultural backgrounds Where both parents are unaffected carriers of the autosomal recessive faulty gene for a particular genetic condition, there is 1 chance in 4 (25% chance) in every pregnancy that their child will inherit the faulty gene copy from both parents and be affected by or predisposed to develop the condition When only one parent is an unaffected carrier of the autosomal recessive faulty gene, there is no chance that their child will be affected by or predisposed to develop the condition Where both parents affected by the condition, they will both have two copies of the autosomal recessive faulty genes. All of their children will also be affected by or predisposed to develop the condition Where one parent is an unaffected carriers of the autosomal recessive faulty gene for a particular genetic condition, and the other parent is affected by the condition, 1 chance in 2 (50% chance) in every pregnancy that they will have a child who inherits both copies of the faulty gene. In this case, the child will be affected or predisposed to develop the condition
Epigenetics is the study of heritable changes in gene expression (active versus inactive genes) that do not involve changes to the underlying DNA sequence — a change in phenotype without a change in genotype — which in turn affects how cells read the genes. - [https://www.whatisepigenetics.com/fundamentals/]
Author of this presentation: The University of Western Australia
THIS PRESENATATION IS FOR THE MEDICAL STUDENTS WHO ALSO HAVE GENETICS AND IF THEY NEED TO GIVE A SEMINAR BASED ON THIS TOPIC THIS PRESENATATION SHALL PROVE USEFUL
Molecular diagnosis of genetic disease ppt for studentsthirupathiSathya
DEFINITION:
Dna analysis can be used for the identification of carriers of hereditary disorders.
For prenatal diagnosis of serious genetic conditions yearly diagnosis before the onset of symptoms is done MOLECULAR DIAGNOSIS OF GENETIC DISEASE
CYSTIC FIBROSIS:
Cystic fibrosis is a genetic disease that affect mostly lungs and also the pancreas.
Screening test:
It is a complex process
Large number of genetic alterations have to be done.
For eg : It is the one of the most common lethal autosomal recessive disorder in Europe.
It is caused by mutations to cystic fibrosis transmembrane conductance regulator(CFTR) gene .
Screening individuals who may be at risk for cystic fibrosis for 500 different mutations is a daunting task.
Diagnosis test that screen for a large number of mutations of a single gene in one assay being developed.
SICKLE CELL ANEMIA:
It is a disorder where red blood cells become rigid and sticky and are shaped like “sickle”.
This irregularly shaped cells stucks in small blood vessels which can slow and block the blood flow and oxygen to all the parts of the body.
There’s no cure for sickle cell anemia.
Screening for sickle cell anemia:
SCA is a genetic disease that is the result of a single nucleotide change in the codon for the sixth aminoacid of the β- chain of the hemoglobin molecule.
The anemia is caused by the inability of the mutated hemoglobin to carry sufficient oxygen.
Target – probe hybridasation is done.
This presentation on Epigenetics is most advanced and evidence based one. Its Very helpful for Genetics students and research fellows, Reproductive Medicine specialist, Reproductive Biologist, Infertility practitioners
Autosomal recessive inheritance refers to the pattern of inheritance of a condition directly or indirectly due to a recessive faulty gene copy located on an autosome Conditions that follow a pattern of autosomal recessive inheritance usually affect men and women equally and include cystic fibrosis, thalassaemia, Tay-Sachs disease and haemochromatosis. These autosomal recessive conditions are more common in individuals of certain ethnic or cultural backgrounds Where both parents are unaffected carriers of the autosomal recessive faulty gene for a particular genetic condition, there is 1 chance in 4 (25% chance) in every pregnancy that their child will inherit the faulty gene copy from both parents and be affected by or predisposed to develop the condition When only one parent is an unaffected carrier of the autosomal recessive faulty gene, there is no chance that their child will be affected by or predisposed to develop the condition Where both parents affected by the condition, they will both have two copies of the autosomal recessive faulty genes. All of their children will also be affected by or predisposed to develop the condition Where one parent is an unaffected carriers of the autosomal recessive faulty gene for a particular genetic condition, and the other parent is affected by the condition, 1 chance in 2 (50% chance) in every pregnancy that they will have a child who inherits both copies of the faulty gene. In this case, the child will be affected or predisposed to develop the condition
Epigenetics is the study of heritable changes in gene expression (active versus inactive genes) that do not involve changes to the underlying DNA sequence — a change in phenotype without a change in genotype — which in turn affects how cells read the genes. - [https://www.whatisepigenetics.com/fundamentals/]
Author of this presentation: The University of Western Australia
OSCE REVISION IN OBSTETRICS AND GYNECOLOGY 2015,NEARLY COVERING COURSE CURRICULUM .Prepared by Dr Manal Behery.Professor of OB&Gyne .Faculty of medicine,Zagazig University
Genetic disease and other inborn errorsMahimaGirase
This ppt is about the diseases and inborn errors of genes in human body.
this ppt includes; genetic epidemiology, genetic research, aim and scope for genetic disorders
Genetic testing (evaluation)
This is a type of clinical test that identifies changes in chromosomes, genes, or proteins. Genetic testing can provide information about a person's genes and chromosomes.
The results of a genetic test can confirm or rule out a suspected genetic condition or help determine a person’s chance of developing or passing on a genetic disorder.
At present, more than 1,000 types of genetic tests are currently in use, and more are being developed.
Methods used for genetic testing:
Molecular genetic tests (or gene tests): It tests single genes or short lengths of DNA to identify variations or mutations that lead to a genetic disorder.
Chromosomal genetic tests: It analyze whole chromosomes or long lengths of DNA to see if there are large genetic changes.
Biochemical genetic tests: It tests the amount or activity level of protein abnormalities to indicate changes in the DNA that result in a genetic disorder.
Genetic testing is voluntary. Because testing has benefits as well as limitations and risks, the decision about whether to be tested is a personal and complex one. A geneticist or genetic counselor can help by providing information about the pros and cons of the test and discussing the social and emotional aspects of testing.
Genomics is the study of the structure and action of the genome, i.e. the sum total of genetic material present in an organism. Genetics is the study of heredity and of the mechanisms by which genetic factors are transmitted from one generation to the next.
Gene therapy involves the insertion of a functioning gene into cells to correct a cellular dysfunction
KEY WORDS : GENETICS, MUTATION , GENETIC ENGINEERING.
In medicine, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease.[ The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.[2] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.
The leaflet aims at providing general objective information on genetic tests, including their nature and the potential implications of their results. It presents the different types of tests available, their applications in the medical field and the extent and limit of the significance of the information resulting from these tests.
More information - www.coe.int/bioethics
INTRODUCTION OF GENE THERAPY, HISTORY OF GENE THERAPY, Process of gene therapy, Methods of gene therapy, Ex vivo gene therapy , In Vivo Gene Therapy , Uses of gene therapy, Target sites for Gene Therapy , Vectors for gene therapy , Viral Vectors, Non Viral Vectors,
Welcome to TechSoup New Member Orientation and Q&A (May 2024).pdfTechSoup
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The Roman Empire, a vast and enduring power, stands as one of history's most remarkable civilizations, leaving an indelible imprint on the world. It emerged from the Roman Republic, transitioning into an imperial powerhouse under the leadership of Augustus Caesar in 27 BCE. This transformation marked the beginning of an era defined by unprecedented territorial expansion, architectural marvels, and profound cultural influence.
The empire's roots lie in the city of Rome, founded, according to legend, by Romulus in 753 BCE. Over centuries, Rome evolved from a small settlement to a formidable republic, characterized by a complex political system with elected officials and checks on power. However, internal strife, class conflicts, and military ambitions paved the way for the end of the Republic. Julius Caesar’s dictatorship and subsequent assassination in 44 BCE created a power vacuum, leading to a civil war. Octavian, later Augustus, emerged victorious, heralding the Roman Empire’s birth.
Under Augustus, the empire experienced the Pax Romana, a 200-year period of relative peace and stability. Augustus reformed the military, established efficient administrative systems, and initiated grand construction projects. The empire's borders expanded, encompassing territories from Britain to Egypt and from Spain to the Euphrates. Roman legions, renowned for their discipline and engineering prowess, secured and maintained these vast territories, building roads, fortifications, and cities that facilitated control and integration.
The Roman Empire’s society was hierarchical, with a rigid class system. At the top were the patricians, wealthy elites who held significant political power. Below them were the plebeians, free citizens with limited political influence, and the vast numbers of slaves who formed the backbone of the economy. The family unit was central, governed by the paterfamilias, the male head who held absolute authority.
Culturally, the Romans were eclectic, absorbing and adapting elements from the civilizations they encountered, particularly the Greeks. Roman art, literature, and philosophy reflected this synthesis, creating a rich cultural tapestry. Latin, the Roman language, became the lingua franca of the Western world, influencing numerous modern languages.
Roman architecture and engineering achievements were monumental. They perfected the arch, vault, and dome, constructing enduring structures like the Colosseum, Pantheon, and aqueducts. These engineering marvels not only showcased Roman ingenuity but also served practical purposes, from public entertainment to water supply.
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Macroeconomics- Movie Location
This will be used as part of your Personal Professional Portfolio once graded.
Objective:
Prepare a presentation or a paper using research, basic comparative analysis, data organization and application of economic information. You will make an informed assessment of an economic climate outside of the United States to accomplish an entertainment industry objective.
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Operation “Blue Star” is the only event in the history of Independent India where the state went into war with its own people. Even after about 40 years it is not clear if it was culmination of states anger over people of the region, a political game of power or start of dictatorial chapter in the democratic setup.
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June 3, 2024 Anti-Semitism Letter Sent to MIT President Kornbluth and MIT Cor...Levi Shapiro
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Dear Dr. Kornbluth and Mr. Gorenberg,
The US House of Representatives is deeply concerned by ongoing and pervasive acts of antisemitic
harassment and intimidation at the Massachusetts Institute of Technology (MIT). Failing to act decisively to ensure a safe learning environment for all students would be a grave dereliction of your responsibilities as President of MIT and Chair of the MIT Corporation.
This Congress will not stand idly by and allow an environment hostile to Jewish students to persist. The House believes that your institution is in violation of Title VI of the Civil Rights Act, and the inability or
unwillingness to rectify this violation through action requires accountability.
Postsecondary education is a unique opportunity for students to learn and have their ideas and beliefs challenged. However, universities receiving hundreds of millions of federal funds annually have denied
students that opportunity and have been hijacked to become venues for the promotion of terrorism, antisemitic harassment and intimidation, unlawful encampments, and in some cases, assaults and riots.
The House of Representatives will not countenance the use of federal funds to indoctrinate students into hateful, antisemitic, anti-American supporters of terrorism. Investigations into campus antisemitism by the Committee on Education and the Workforce and the Committee on Ways and Means have been expanded into a Congress-wide probe across all relevant jurisdictions to address this national crisis. The undersigned Committees will conduct oversight into the use of federal funds at MIT and its learning environment under authorities granted to each Committee.
• The Committee on Education and the Workforce has been investigating your institution since December 7, 2023. The Committee has broad jurisdiction over postsecondary education, including its compliance with Title VI of the Civil Rights Act, campus safety concerns over disruptions to the learning environment, and the awarding of federal student aid under the Higher Education Act.
• The Committee on Oversight and Accountability is investigating the sources of funding and other support flowing to groups espousing pro-Hamas propaganda and engaged in antisemitic harassment and intimidation of students. The Committee on Oversight and Accountability is the principal oversight committee of the US House of Representatives and has broad authority to investigate “any matter” at “any time” under House Rule X.
• The Committee on Ways and Means has been investigating several universities since November 15, 2023, when the Committee held a hearing entitled From Ivory Towers to Dark Corners: Investigating the Nexus Between Antisemitism, Tax-Exempt Universities, and Terror Financing. The Committee followed the hearing with letters to those institutions on January 10, 202
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Introduction to AI for Nonprofits with Tapp Network
Genetic screening & gene therapy
1. GENETIC SCREENING & GENE THERAPY Genetic screening & Gene therapy Dr. Dinesh T Junior resident, Department of Physiology, JIPMER Dr sclerodinesh
2. Introduction
3.
4. History Technology to detect and treat inborn diseases - 1961. 1972 Friedmann and Roblin authored a paper in Science titled "Gene therapy for human genetic disease?“ The late 1980's, an international team of scientists began the project to map the human genome. September 14, 1990 - first approved gene therapy case in the United States took place.
5. 1992 - Doctor Claudio Bordignon, Milan, Italy performed the first procedure of gene therapy using hematopoietic stem cells. 1995 DNA testing in forensic cases gains fame in the O.J. Simpson trial. 2002 - first successful gene therapy treatment for adenosine deaminase-deficiency (SCID) 2003 – at University of California, Los Angeles research team inserted genes into the brain using liposome coated in a polymer called polyethylene glycol
6. 2006 - Preston Nix from the University of Pennsylvania School of Medicine reported on VRX496, a gene-based immunotherapy for the treatment of human immunodeficiency virus (HIV) that uses a lentiviral vector for delivery of an antisense gene against the HIV envelope 2007 – Moorfields Eye Hospital and University College London's Institute of Ophthalmology announced the world's first gene therapy trial for inherited retinal disease 2008 there were more than 1,200 clinically applicable genetic tests available.
8. What is genetic screening? The newest and most sophisticated of the techniques used to test for genetic disorders. One of the fastest moving fields in medical science. A technique to determine the genotype or phenotype of an organism. Determines risk of having or passing on a genetic disorder.
9. Genetic screening Genetic screening is often used to detect faulty or abnormal genes in an organism Can detect some genes related to an increasedrisk of cancer Can detect some genes known to cause geneticdisorders
10. Genetic tests The analysis of chromosomes (DNA), proteins, and certain metabolites in order to detect heritable disease-related genotypes, mutations, phenotypes, or karyotype for clinical purposes.
11. Gene tests (also called DNA-based tests), in a broader sense Direct examination of the DNA molecule Biochemical tests for such gene products as enzymes and other proteins Microscopic examination of stained or fluorescent chromosomes
12. Genetic tests Who can order? What are the samples needed? How to interpret the tests? What are all the risks? Ethical considerations?
26. Family history of neural tube defect or other congenital abnormalities
27. Newborn Screening Tests: Maple Syrup Urine Disease Congenital Adrenal Hyperplasia Congenital Hypothyroidism Glactosemia Biotinidase Deficiency Homocystinuria Phylketonuria (PKU) Sickle cell and Other Hemoglobinopathies
28. Pre implantation Genetic Diagnosis (PGD) Pre implantation Genetic Diagnosis (PGD) uses in vitro fertilisation (IVF) to create embryos. Tests one or two cells from each embryo for a specific genetic abnormality. Identifies unaffected embryos for transfer to the uterus. The approach through PGD assists couples at risk of an inherited disorder to avoid the birth of an affected child without going through selective pregnancy termination.
29.
30. To avoid having children with devastating diseases
45. Gene therapy is the replacement of faulty genes. Introduction of functional genetic material into target cells to replace or supplement defective genes, or to modify target cells so as to achieve therapeutic goals.
46. In theory it is possible to transform either somatic cells (most cells of the body) or cells of the germ line (such as sperm cells,ova, and their stem cell precursors).
47.
48.
49. Somatic Cell Therapy This is when a gene is introduced into a patient to help them recover from a disease.
50. Germ Line Therapy Changes are made to genes that will affect subsequent generations.
51. Applications of Gene Therapy Radical cure of single gene diseases e.g. cystic fibrosis, haemoglobinopathies. Amelioration of diseases with or without a genetic component e.g. malignancies, neurodegenerative diseases, infectious diseases.
53. Vectors in gene therapy: Non-viral methods Viruses Adeno-associated viruses Retroviruses Oligonucleotides Hybrid methods Lipoplexes and polyplexes Adenoviruses Naked DNA Envelope protein pseudotyping of viral vectors
54.
55. Gene therapy using an adenovirus vector. A new gene is inserted into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.
56. Non viral vectors Un complexed plasmid DNA DNA coated gold particles Liposomes DNA – protein conjugates
58. Un complexed Plasmid DNA Purified DNA or mRNA injected directly into tissues Injected into muscle and skin • Utility in immunization/ vaccination against Infectious diseases • Ectopic synthesis of therapeutic proteins as erythropoietin.
63. DNA- Protein conjugates • Cell- specific DNA delivery systems • Utilize unique cell surface receptors on target cells • Chemical cross linking methods used
64.
65. Gene Transfer techniques • In vivo Suspension containing vector is injected directly into the patient either systemically (i.v.) or directly into target tissue (e.g. malignant tumour) • Ex vivo Target cells (stem cells,myoblasts,fibroblasts etc) removed from the patient, treated with vector and injected back into the patient
66.
67.
68.
69. Spectrum of gene expression Gene replacement for single gene disorders Gene repair Gene inactivation Ectopic synthesis of therapeutic proteins Cancer gene therapy
70. A) Immunodeficiency Disorders Adenosine Deaminase Deficiency X- linked SCID Chronic Granulomatous disease B) Liver Disease Familial Hypercholesterolemia Haemophilia A Target diseases