Gene therapy involves inserting normal genes into patients to replace abnormal genes that cause disease. It is being studied for many diseases like immunodeficiencies, hemophilia, Parkinson's, and cancer. The first gene therapy occurred in 1990 and involved treating a genetic immune deficiency. While it offers potential cures, there are also risks and ethical concerns around its use.
Gene therapy have the potential to revolutionize the practice of medicine. A breakthrough may come anytime and a day may come when almost every disease will have a gene therapy.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
These slide include gene therapy defines with their types like Germ line gene therapy,Somatic gene therapy.
with Need of Gene therapy
strategies of gene therapy
Methods of Gene transfer & with
GENE THERAPY FOR INHERITED DISORDERS
this helps to understand the normal techniques related to biotechnology in a simple manner and provides you broad idea about the subject. A brief knowledge about the topic is presented in this presentation.
Gene therapy have the potential to revolutionize the practice of medicine. A breakthrough may come anytime and a day may come when almost every disease will have a gene therapy.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
These slide include gene therapy defines with their types like Germ line gene therapy,Somatic gene therapy.
with Need of Gene therapy
strategies of gene therapy
Methods of Gene transfer & with
GENE THERAPY FOR INHERITED DISORDERS
this helps to understand the normal techniques related to biotechnology in a simple manner and provides you broad idea about the subject. A brief knowledge about the topic is presented in this presentation.
Production and purification of Viral vectors for gene and cell therapy appli...Dr. Priyabrata Pattnaik
Presentation at "2016 Osong BioExcellence - Renaissance in Immunotherapy" at South Korea, an event jointly hosted by Kbio Health and Merck on 6th October 2016.
Fluorescence- Activated Cell Sorter is a powerful technique used in cell sorting, cell-cycle analysis etc.
The presentation gives a basic understanding of the principle of FACS, instrumentation, interpretation of results, applications, how to do cell-cycle analysis using FACS and various troubleshooting tips.
Please share this webinar with anyone who may be interested!
Watch all our webinars: https://www.youtube.com/playlist?list=PL4dDQscmFYu_ezxuxnAE61hx4JlqAKXpR
Cancer care is increasingly tailored to individual patients, who can undergo genetic or biomarker testing soon after diagnosis, to determine which treatments have the best chance of shrinking or eliminating tumours.
In this webinar, a pathologist and clinical oncologist discuss:
● how they are using these new tests,
● how they communicate results and treatment options to patients and caregivers, and
● how patients can be better informed on the kinds of tests that are in development or in use across Canada
View the video: https://youtu.be/_Wai_uMQKEQ
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Viral Based Gene Delivery System for Car-t Cell Engineering Creative-Biolabs
A brief introduction about lentiviral vector gene delivery system and its application in CAR-T cell construction. Creative Biolabs offers high quality lentivirus based CAR gene delivery service to help with your CAR-T cell development projects.
Gene therapy is an experimental treatment that involves introducing genetic material into a person’s cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinson's disease, cancer and even HIV, through a number of different approaches (see video: 'Gene Therapy a new tool to cure human diseases'). A gene can be delivered to a cell using a carrier known as a “vector.” The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy, but it has been used with some success.
Buying a refrigerator freezer combo isn’t the easiest thing in the world. First, most companies often have a poor selection. Other companies may overcharge for poor-quality products. Most commercial display coolers manufactures also don’t offer an adequate warranty policy to insure your investment. If you’re looking for a new commercial cooler or a refrigerator freezer combo for your business, then you should look no further than http://srcrefrigeration.com/
Slide deals with the brief explanation on refrigeration which is one of the most important application of our lives.
The slide will be helpful for students studying Basic Mechanical engineering.
Recombinant viral vectors are genetic engineering tools commonly used for gene transfer purpose with high transfection efficiency and site specific gene insertion.
NUCLEIC ACID BASED THERAPEUTIC DELIVERY SYSTEM by pramesh..pptxPRAMESHPANWAR1
Name of the title: Nucleic Acid-Based Therapeutic Delivery System.
It includes information about nucleic acid, gene therapy, and its type, a method to deliver the desired DNA, i.e., vectors and their types, with proper examples and diagrams, and how these things help in delivering a nucleic acid-based therapeutic drug delivery system.
Assalamualaikum
Hope you all are fine and doing well. Submitting my work on gene therapy. Let me know you this is a basic knowledge assignment and I have written it for students use and I hope it will be helpful for you. Keep me remember in your prayers.
Thanks.
Gene therapy is the process of inserting therapeutic genes into cells to prevent or cure wide range of diseases. The newly introduced genes will encode proteins and correct the deficiencies that occur in genetic diseases. Gene therapy primarily involves genetic manipulations in animals or humans to correct a disease, and keep organism in good health. It is a technique for correcting defective genes responsible for disease and development.
Definition, Gene therapy, types of gene therapy, germline gene therapy, somatic cell gene therapy, basic process of gene therapy and potential targets for gene therapy.
Explore natural remedies for syphilis treatment in Singapore. Discover alternative therapies, herbal remedies, and lifestyle changes that may complement conventional treatments. Learn about holistic approaches to managing syphilis symptoms and supporting overall health.
New Directions in Targeted Therapeutic Approaches for Older Adults With Mantl...i3 Health
i3 Health is pleased to make the speaker slides from this activity available for use as a non-accredited self-study or teaching resource.
This slide deck presented by Dr. Kami Maddocks, Professor-Clinical in the Division of Hematology and
Associate Division Director for Ambulatory Operations
The Ohio State University Comprehensive Cancer Center, will provide insight into new directions in targeted therapeutic approaches for older adults with mantle cell lymphoma.
STATEMENT OF NEED
Mantle cell lymphoma (MCL) is a rare, aggressive B-cell non-Hodgkin lymphoma (NHL) accounting for 5% to 7% of all lymphomas. Its prognosis ranges from indolent disease that does not require treatment for years to very aggressive disease, which is associated with poor survival (Silkenstedt et al, 2021). Typically, MCL is diagnosed at advanced stage and in older patients who cannot tolerate intensive therapy (NCCN, 2022). Although recent advances have slightly increased remission rates, recurrence and relapse remain very common, leading to a median overall survival between 3 and 6 years (LLS, 2021). Though there are several effective options, progress is still needed towards establishing an accepted frontline approach for MCL (Castellino et al, 2022). Treatment selection and management of MCL are complicated by the heterogeneity of prognosis, advanced age and comorbidities of patients, and lack of an established standard approach for treatment, making it vital that clinicians be familiar with the latest research and advances in this area. In this activity chaired by Michael Wang, MD, Professor in the Department of Lymphoma & Myeloma at MD Anderson Cancer Center, expert faculty will discuss prognostic factors informing treatment, the promising results of recent trials in new therapeutic approaches, and the implications of treatment resistance in therapeutic selection for MCL.
Target Audience
Hematology/oncology fellows, attending faculty, and other health care professionals involved in the treatment of patients with mantle cell lymphoma (MCL).
Learning Objectives
1.) Identify clinical and biological prognostic factors that can guide treatment decision making for older adults with MCL
2.) Evaluate emerging data on targeted therapeutic approaches for treatment-naive and relapsed/refractory MCL and their applicability to older adults
3.) Assess mechanisms of resistance to targeted therapies for MCL and their implications for treatment selection
Report Back from SGO 2024: What’s the Latest in Cervical Cancer?bkling
Are you curious about what’s new in cervical cancer research or unsure what the findings mean? Join Dr. Emily Ko, a gynecologic oncologist at Penn Medicine, to learn about the latest updates from the Society of Gynecologic Oncology (SGO) 2024 Annual Meeting on Women’s Cancer. Dr. Ko will discuss what the research presented at the conference means for you and answer your questions about the new developments.
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Ve...kevinkariuki227
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
TEST BANK for Operations Management, 14th Edition by William J. Stevenson, Verified Chapters 1 - 19, Complete Newest Version.pdf
Pulmonary Thromboembolism - etilogy, types, medical- Surgical and nursing man...VarunMahajani
Disruption of blood supply to lung alveoli due to blockage of one or more pulmonary blood vessels is called as Pulmonary thromboembolism. In this presentation we will discuss its causes, types and its management in depth.
- Video recording of this lecture in English language: https://youtu.be/lK81BzxMqdo
- Video recording of this lecture in Arabic language: https://youtu.be/Ve4P0COk9OI
- Link to download the book free: https://nephrotube.blogspot.com/p/nephrotube-nephrology-books.html
- Link to NephroTube website: www.NephroTube.com
- Link to NephroTube social media accounts: https://nephrotube.blogspot.com/p/join-nephrotube-on-social-media.html
These simplified slides by Dr. Sidra Arshad present an overview of the non-respiratory functions of the respiratory tract.
Learning objectives:
1. Enlist the non-respiratory functions of the respiratory tract
2. Briefly explain how these functions are carried out
3. Discuss the significance of dead space
4. Differentiate between minute ventilation and alveolar ventilation
5. Describe the cough and sneeze reflexes
Study Resources:
1. Chapter 39, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 34, Ganong’s Review of Medical Physiology, 26th edition
3. Chapter 17, Human Physiology by Lauralee Sherwood, 9th edition
4. Non-respiratory functions of the lungs https://academic.oup.com/bjaed/article/13/3/98/278874
These lecture slides, by Dr Sidra Arshad, offer a quick overview of physiological basis of a normal electrocardiogram.
Learning objectives:
1. Define an electrocardiogram (ECG) and electrocardiography
2. Describe how dipoles generated by the heart produce the waveforms of the ECG
3. Describe the components of a normal electrocardiogram of a typical bipolar leads (limb II)
4. Differentiate between intervals and segments
5. Enlist some common indications for obtaining an ECG
Study Resources:
1. Chapter 11, Guyton and Hall Textbook of Medical Physiology, 14th edition
2. Chapter 9, Human Physiology - From Cells to Systems, Lauralee Sherwood, 9th edition
3. Chapter 29, Ganong’s Review of Medical Physiology, 26th edition
4. Electrocardiogram, StatPearls - https://www.ncbi.nlm.nih.gov/books/NBK549803/
5. ECG in Medical Practice by ABM Abdullah, 4th edition
6. ECG Basics, http://www.nataliescasebook.com/tag/e-c-g-basics
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Gene therapy ppt
1. GENE THERAPY
Presented by:
Nurul Miza Shasheiha binti Abdul Mutalib -5089-
Wan Fathiah Nasuha binti Wan Nudri -5117-
Nurul Husna binti Muryadi -5088-
Nur Amalin Shafirah binti
Nur Farzana Sahirah binti Ariffuddin
2. WHAT IS GENE THERAPY ?
• Definiton: an experimental technique for correcting defective
genes that are responsible for disease development
• The most common form of gene therapy involves inserting a
normal gene to replace an abnormal gene
• Other approaches used:
Replacing a mutated gene that causes disease with a
healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is
functioning improperly.
Introducing a new gene into the body to help fight a disease.
3. • Researchers are studying gene therapy for a number of
diseases, such as
Severe combined immuno-deficiencies (SCID)
Hemophilia
Parkinson's disease
Cancer
HIV
4. HISTORY AND DEVELOPMENT
OF GENE THERAPY
• 1960: The concepts of Gene Therapy was introduced
• 1970: Friedmann and Roblin author of a paper in Science titled "Gene
therapy for human genetic disease?” cite the first attempt to perform
gene therapy
• 1990:
The first approved gene therapy case at the National Institute of
Health, U.K. It was performed on a four year old girl named
Ashanti DaSilva. It was a treatment for a genetic defect that left
her with an immune system deficiency
New gene therapy approach repairs errors in messenger RNA
derived from defective genes. This technique has the potential to
treat the blood disorder Thalassaemia, Cystic fibrosis, and some
cancers
Sickle cell disease is successfully treated in mice
5. • 1992: Doctor Claudio Bordignon working at the Vita-Salute San Raffaele
University, Milan, Italy performed the first procedure of gene therapy using
hematopoietic stem cells as vectors to deliver genes intended to correct
hereditary diseases
• 1999: Death of Jesse Gelsinger in a gene-therapy experiment resulted in a
significant setback to gene therapy research in the United States
• 2006: Scientists at the National Institutes of Health (Bethesda, Maryland) have
successfully treated metastatic melanoma in two patients. This study constitutes
one of the first demonstrations that gene therapy can be effective in treating
cancer.
• 2007- 2011: Research is still ongoing and the number of diseases that has been
treated successfully by gene therapy increases.
Retinal disease
Colour blindness
Adrenoleukodystrophy
• 2011: Medical community accepted that it can cure HIV as in 2008, Gero Hutter
has cured a man from HIV using gene therapy
6.
7. Result in permanent changes.
Potential for offering a permanent therapeutic
effect for all who inherit the target gene.
Possibility of eliminating some diseases from a
particular family.
Also raises controversy:
Some people view this type of therapy as
unnatural, and liken it to "playing God”.
Others have concerns about the technical
aspects.
8.
9. Affects only the targeted cells in the
patient, and is not passed to future
generations.
Short-lived because the cells of most tissues
ultimately die and are replaced by new
cells.
Transporting the gene to the target cells or
tissue is also problematic.
Appropriate and acceptable for many
disorders, including cystic fibrosis, muscular
dystrophy, cancer, and certain infectious
diseases.
10.
11.
12.
13. BASIC PROCESS OF
GENE THERAPY
1. VIRAL VECTOR
2. NON VIRAL VECTOR
14. GT utilizes the delivery of DNA into cells, which can be
accomplished by a number of methods.
The two major classes of methods :
recombinant viruses – VIRAL VECTOR
naked DNA or DNA complexes – NONVIRAL
VECTOR
15. Viruses have evolved a way of encapsulating and
delivering their genes to human cells in a pathogenic
manner. Scientists have tried to harness this ability by
manipulating the viral genome to remove disease-
causing genes and insert therapeutic ones .
VIRAL VECTOR
16. Virus bind to their hosts and introduce their genetic
material into the host cell.
Plausible strategy for gene therapy, by removing the
viral DNA and using the virus as a vehicle to deliver
the therapeutic DNA.
The viruses used are altered to make them
safe, although some risks still exist with gene
therapy.
VIRUS
17. Many GT clinical trials rely on retroviruses or
adenoviruses to deliver the desired gene.
Other viruses used as vectors include adeno-associated
viruses, lentiviruses, pox viruses, alphaviruses, and
herpes viruses.
Differ in how well they transfer genes to the cells they
recognize and are able to infect, and whether they alter
the cell’s DNA permanently or temporarily
TYPES OF VIRUS
18.
19.
20. Are a tool commonly used by molecular biologists to
deliver genetic material into cells.
Can be performed in vivo or in vitro.
Viruses have evolved specialized molecular
mechanisms to efficiently transport their genomes
inside the cells they infect.
Delivery of genes by a virus is termed transduction
and the infected cells are described as transduced.
VIRAL VECTOR
21.
22. Methods of non-viral gene delivery have also been
explored using physical (carrier-free gene delivery)
and chemical approaches (synthetic vector-based
gene delivery).
NON VIRAL VECTOR
23. Physical approaches, including
Needle injection
Electroporation
Gene gun
Ultrasound
Hydrodynamic delivery
employ a physical force that permeates the cell
membrane and facilitates intracellular gene transfer
PHYSICAL METHOD
24. The simplest method of non-viral transfection.
Clinical trials carried out of intramuscular injection
of a naked DNA plasmid have occurred with some
success; however, the expression has been very low
in comparison to other methods of transfection.
I. NAKED DNA
25. This success, however, does not compare to that of
the other methods, leading to research into more
efficient methods for delivery of the naked DNA
such as electroporation and the use of a "gene gun",
which shoots DNA coated gold particles into the cell
using high pressure gas.
26. CHEMICAL METHODS
THAT ENHANCE THE
DELIVERY OF GENE THERAPY
-lipoplexes & polyplexes-
27. DNA must be protected from damage & its entry
into the cell must be facilitated
Plasmid DNA can be covered with lipids in an
organized structure like a micelle or a
liposome complexed with DNA it is called a
lipoplex
3 types of lipids:
anionic (negatively charged)
neutral
cationic (positively charged)
LIPOPLEXES
28. Initially, anionic and neutral lipids :
-were used for the construction of lipoplexes for
synthetic vectors.
-but,there is little toxicity associated with them,
-they are compatible with body fluids
-there was a possibility of adapting them to be
tissue specific
-they are complicated turned to the cationic
versions.
Cationic lipids, due to their positive charge,
-naturally complex with the negatively
charged DNA.
-their charge they interact with the cell membrane
-endocytosis of the lipoplex occurs
-DNA is released into the cytoplasm.
-The cationic lipids also protect against
degradation of the DNA by the cell.
29. Common used of lipoplexes
In gene transfer into cancer cells, where the
supplied genes have activated tumor
suppressor control genes in the cell
decrease the activity of oncogenes.
useful in transfecting respiratory epithelial cells,
so they may be used for treatment of genetic
respiratory diseases such as cystic fibrosis.
30. Complexes of polymers with DNA are called
polyplexes
consist of cationic polymers and their production
is regulated by ionic interactions.
large difference compared to lipoplexes is that
polyplexes cannot release their DNA load into
the cytoplasm,
End= co-transfection with endosome-lytic
agents such as inactivated adenovirus must
occur (to lyse the endosome that is made during
endocytosis, the process by which the polyplex
enters the cell)
POLYPLEXES
32. GENE THERAPY CURES
BLINDNESS
Cure blindness of inherited condition
Leber’s conginetal amaurosis
- inherited disease caused by an
abnormality in a gene called RPE65.
- The condition appears at birth or in the first
few months of life and causes progressive
worse and loss of vision.
33. HOW IT WORKS??
used harmless viruses
enable access to the cells beneath the
retinas of patients
By using a very fine needle
-safe in an extremely fragile tissue and can
improve vision in a condition previously
considered wholly untreatable.
• http://www.youtube.com/watch?v=d_YJZn-ft_Q
35. GENE THERAPY REDUCES
PARKINSON’S DISEASE SYMPTOMS
it significantly improved the weakness of the
symptoms such as tremors, motor skill problems, and
rigidity
Main- overactive brain region: the subthalamic
nucleus should be introduced with gene
that would produce GABA—an inhibitory
chemical—then they could potentially quiet that
brain region and alleviate tremors.
36. HOW IT WORKS??
Done with local anesthesia, used a harmless,
inactive virus [AAV-2 GAD]
Deliver the GAD gene into patient’s
subthalamic nucleus
The gene instructs cells to begin making
GABA neurotransmitters to re-establish the
normal chemical balance that becomes
dysfunctional as the disease progresses
37. ADVANTAGES OF GENE THERAPY
• Give a chance of a normal life to baby born with
genetic disease.
• Give hope of healthy life to cancer patient.
• For certain disease that do not have any cure
except gene therapy, it could save many lives
38. DISADVANTAGES OF
GENE THERAPY
• The genetic testing, screening and research in finding the
availability of certain gene is very controversy.
• May increase rate of abortion if prenatal test regarding baby
with genetic disease is done.
• The cost is very high and the patient might need an insurance to
cover the treatment.
• Cosmetic industry may monopolized this gene therapy if it is
used in enhancing beauty and in vanishing the aging
effect, rather than used for treatment of a disease.
39. ETHICAL QUESTIONS SURROUNDING
GENE THERAPY
• How can “good” and “bad” uses of gene therapy be
distinguished?
• Who decides which traits are normal and which constitute
a disability or disorder?
• Will the therapy only benefit the wealthy due to its high
cost?
• Could the widespread use of gene therapy make the
society less accepting of people who are different?
• Should people be allowed to use gene therapy to enhance
basic human traits such as height, intelligence, or athletic
ability?