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Gene Therapy: Past, Present, and Future

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This program will include a discussion of off-label treatment and investigational agents not approved by the FDA for use in the US, and data that were.

Immunotherapies: Key Considerations in the Identification and Management of irAEs.

Systemic Lupus Erythematosus

A New Path Forward: Immune Checkpoint Inhibitors in Bladder Cancer

Clinical Trials in IBD.

Why Gene Therapy for Hemophilia?

This program will include a discussion of off-label treatment and investigational agents not approved by the FDA for use in the United States.

Unraveling Clinical Developments in NASH

Advances in Managing Inhibitors in Patients With Hemophilia A

Pseudomonas Lung Infections in Cystic Fibrosis

Dual Antiretroviral Therapy

Progression After Cancer Immunotherapy in Advanced NSCLC

New Standards of Care in ALK-Translocated Advanced NSCLC

Hemophilia Updates: Incorporating New Concepts Into Practice

Updates in Management of Atopic Dermatitis From Real Patient Cases

Mid-Year Hemophilia Update

Induction Chemotherapy for Patients With High-Risk or Secondary AML

Determining the Best Treatment Algorithm for Patients With Head and Neck Cancer.

The Resurgence of Topical Treatments Across a Spectrum of Psoriasis

Chronic Idiopathic Urticaria

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Gene Therapy PPT | Free Download Presentation

Gene Therapy PPT | Free Download Presentation : Hello friends, today we are going to share a complete presentation on gene therapy. gene therapy is a technique that is used to cure or treat disease by modifying a person’s genes. There are several mechanisms by which gene therapy works.

Also See: General Seminar Topics

• Bringing in new genes in a person’s body to treat a disease.
• Making genes inactive that are not functioning properly.
• Completely replacing a gene (that is causing the disease) with a fresh and healthy copy of a gene.

Lots of studies and experiments have been made into this technique and it is growing day by day as it is treating many ill peoples. The very first approved gene therapy on humans was done in 1990 to sure a disease named SCID (Severe Combined Immunodeficiency).  So lets us learn everything about gene therapy in the above ppt and at the end of this post, you will get a full gene therapy ppt for free download.

Also See: Non- Technical Seminar Topics

There are basically two types of gene therapies that are discussed below.

• Types of Gene Therapy

In Vivo Gene Therapy : In this kind of gene therapy, genes are directly delivered into the target cell to sure a missing gene.

Ex Vivo Gene Therapy : In this gene therapy, genes are removed from the person’s body, and then they are transplanted back into the person’s body. Blood and immunological diseases are treated with the help of ex vivo gene therapy and it is now widely used therapy.

Also See: Shock PPT

Issues Related to Gene Therapy

• Who decides what is normal and what is disorder? It is questionable.
• Due to its high cost, it is only available for wealthy persons.

Content of Gene Therapy ppt for free download

• What Genes can do
• Why Genetic Disorders
• Law of Inheritance
• What is Gene Therapy
• How It Works
• Gene Therapy is Experimental
• Uses of gene therapy
• Goals of gene therapy
• Future of gene therapy

Also See: Hypertension PPT

Here we are giving you Gene Therapy Seminar and PPT with PDF report. All you need to do is just click on the download link and get it.

Gene Therapy PPT and Seminar Free Download

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Gene therapy.pptx

Related Papers

Shashi Pratap Singh

Dr. Ravindra B. Malabadi

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Gene therapy concept has been being overcome massive challenges from 1972 in ethical, socio-economical and developmental issues. In this review, we have attempted to go through almost all the arenas and described in a methodical way that reflects not only the initial ethical and scientific thoughts but also adorned a solid depiction of gene therapy related physico-chemical barriers, approaches and strategies till to date.

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Gene therapy is an experimental technique that uses genes encoding the expression of proteins that are either endogenous or biological to treat or prevent a disease by inserting it into patient's cells. Previously the challenges faced were the gene titers which then changed to their delivery and now expression of the genes. Initially the trial of gene therapy for adenosine deaminase deficiency had proven to be successful but till date only alipogenetiparvovec has been approved in Europe for lipoprotein lipase deficiency. Recent techniques with adenovirus, r-adeno associated virus (r-aav), retrovirus,herpes simplex virus vectors and non-viral lipoplexes (for transfer of large biological products) have completely revolutionized the future of gene therapy. With the upcoming advances in the field of cancer therapy such as herpes simplex thymidine kinase (hs-tk) and its role in suicidal gene therapy in the treatment of cancers has stretched new horizons in the field of gene therapy. Recently the newer treatment approaches with RNA interference and anti-sense oligonucleotides in muscular dystrophies were certainly encouraging for the researchers to further work on the newer prospects of gene therapy. Gene therapy is currently investigated for cardiac failure and for prevention of brain ageing in Alzheimer's disease.

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Gene Therapy

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Grab our Gene Therapy presentation template for MS PowerPoint and Google Slides to describe the medical approach that genetically modifies an individual’s cells to treat or prevent diseases. Save loads of time and effort with these pre-designed slides!

Healthcare professionals will find this proficiently designed set handy in depicting the objectives, applications, advantages, and disadvantages of gene therapy in a comprehensible manner. You can also explain the features and key aspects related to somatic and germline gene therapy. Moreover, using our PPT, you can shed light on the problems and issues that arise as a result of gene therapy.

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Gene Therapy

Oct 23, 2014

850 likes | 2.53k Views

Gene Therapy. Meredith Calhoun. What is Gene Therapy?. - Gene therapy is a form of treatment that changes defective genes to stop a disease. - Gene therapy can be used instead of medicines or surgery. - It uses viruses to genetically modify a cells DNA. How does it work?.

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Presentation Transcript

Gene Therapy • Meredith Calhoun

What is Gene Therapy? - Gene therapy is a form of treatment that changes defective genes to stop a disease. - Gene therapy can be used instead of medicines or surgery. - It uses viruses to genetically modify a cells DNA.

How does it work? • In gene therapy, researchers use viruses to correct the genes in cells by injecting desired genetic material in to the virus. MANY viruses are then put in the body to deliver the correct gene to the cells or take the cells out and let the DNA be corrected. Then corrected cells are put back into the body. These viruses have been genetically engineered so that they can't reproduce. Scientists are researching other ways to accomplish gene therapy. They are looking at a fatty molecule known as a liposome and physically inserting genes into the cell through "gene guns" or micropipettes.

Steps: • 1. Diagnose disease or disorder and then find the gene that causes the disorder. • 2. Engineer virus and insert desired genes. • 3. Inject viruses into body (Vivo) or remove target cells for the disease (Ex Vivo). • 4. Allow viruses to inject genes into cells DNA to fix disorder. • 5. If target cells were removed and placed in culture with the viruses, then the corrected cells will be placed back in the body. Adenovirus

Two types: • Somatic cell gene therapy- is gene therapy, or changing genes to fix a disease, in normal body cells. • This can fix the disease and cause the correct protein to be made, but the gene will not be passed on to your offspring. • Germline Therapy- is just replacing genes in reproductive cells • Because genes are changed in reproductive cells the new gene can be passed on to the offspring. • Affects many generation's DNA make up. The child will have the correct gene instead of the defected one.

History • The 1960's and 1970's was when the idea of gene therapy first arose. • By 1972, Theodore Friedman and Richard Roblin published a paper explaining Stanfield Rogers gene therapy idea for curing humans with diseases. • In 1985 Dr.s W. French Anderson and Michael Blaese from the National Heart, Lung, and Blood Institute and the National Cancer Institute worked together to find that they could cure people,with Adenosine Deaminase Deficiency (ADA), using gene therapy. They conducted the first gene therapy in ADA cells in a tissue culture.

History Continued • In 1986 Anderson and Blaese transferred correct genes into bone marrow of animals. • In 1988 they found gene therapy was more successful in white blood cells. • In 1989 Anderson and Blaese teamed up with Dr. Steven Rosenberg to see if gene therapy would be successful in patients with cancer, particularly melanoma.

In the 1990's the first condition in humans was treated with Gene Therapy by Dr.s W. French Anderson and Michael Blaese. Two little girls, a four and nine year old, with an immune deficiency called ADA were cured through gene therapy.

Obviously it's a positive that gene therapy can eliminate disorders before they begin or get to bad. • It can guarantee a cure for future suffering of some diseases. • Gene Therapy is considered a "medicine for the future" because it can control or alter Germ cells. Resulting in cured hereditary diseases and changing DNA for future generations. • Possibilities are endless. Since gene therapy is such a new technology, researchers are still finding gene therapy can cure many diseases. • Can possibly cure cancer, heart and lung disease, muscular dystrophy, blindness, AIDS, or even Alzheimer's.

The Cons • With all new technologies there are possible consequences.

Before gene therapy begins,your immune system must be weakened, so that the viruses can correctly inject the gene. The immune system can't tell the difference between a bad virus and a helping virus. • Since it is so new, it can be very expensive. • It's a new technology so not much is known about it. There could be consequences we don't know about yet. • The virus may change DNA in cells that don't need fixings or insert genes into the wrong part of the genome, this can lead to more serious problems such as cancer.

Ethical and Religious Considerations • The first ethical issue is, Gene Therapy would put human fate in our hands by giving us the ability to affect genes of future generations. • Some are concerned it could give people the ability to make a superior race(eugenics), however this is not the goal of geneticists. • Some predict it would be hard to reinforce laws on gene therapy. And it could be offered on the black market. • Religion also plays a large role in an individual's considerations, some consider gene therapy is sinful. People believed gene therapy would be like "playing God".

Current Applications • Gene therapy can be used to correct single gene mutation diseases like: • Cystic Fibrosis • Sickle Cell Anemia • Hemophilia has been treated in the U.S. Corrected the gene that allows the blood to correctly clot. • X-SCID is a immune deficiency where people lack B and T cells. Gene therapy corrects mutation on the X-gene and this causes the cells to be produced. • Type I Diabetes has been corrected in rats and dogs. • Paget's Diseases is a rare bone metabolism disorder caused by a gene being missing.

Current Applications Continued • Chronic Granulomatus Disorder (CDG) has been cured in two patients. This disorder causes people to not be able to fight off bacterial and fungal infections. • Some cancer cells have been cured in a lab with many different types of gene therapy. • Neurodegenerative diseases like Parkinson's and Huntington's Disease have been cured in animals through gene therapy. • Diseases that are caused by more then one gene are being researched but are not yet used because of how complex it is to correct more than one gene at a time.

Case Study • Using Gene Therapy to reverse blindness in dogs.

Curing Blindness in Dogs • University of Pennsylvania researchers led scientists in using gene therapy to cure three puppies that were born blind. • The dogs in this experiment had a disease known as Leber Congenital Amaurosis (LCA). • The gene that is mutated stops making proteins that allow normal vision. • Retinitis Pigmentosa GTPase Regulator (RPGR) is the gene that contains the error. • The protein that can't be produced is photoreceptor ciliary. • The retina deteriorates and becomes dysfunctional when the dog has LCA.

Curing Blindness Continued • A few weeks after the new genes were injected into a retina of one eye in each puppy, they began to gain vision. They could see and interact with humans and began to maneuver around objects. • A month after the genes had been injected into the retina, the scientists began testing the activity of each eye (with or without the corrected genes). • They used a technique called electro retinography, it measures the levels of electrical activity in the retina. • At the two month mark there was a 35%-40% electrical activity occurring in the retina that received gene therapy. The eye that didn't receive gene therapy had little to no activity.

Since LCA occurs in humans, scientists are now researching to see if the gene therapy used on the puppies would still be successful on humans.

Resources • http://www.esgct.eu/useful-information/introduction-to-gene-therapy.aspx • http://ghr.nlm.nih.gov/handbook/therapy/genetherapy • http://www.ndsu.edu/pubweb/~mcclean/plsc431/students98/fleck.htm • http://www.ama-assn.org//ama/pub/physician-resources/medical-science/genetics-molecular-medicine/current-topics/gene-therapy.page • http://history.nih.gov/exhibits/genetics/sect4.htm#1 • http://abcnews.go.com/GMA/t/story?id=127024&ref=https%3A%2F%2Fwww.google.com%2F • http://www.hc-sc.gc.ca/sr-sr/biotech/about-apropos/gen_therap-eng.php

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Realising gene therapy: a biomedical challenge

In recent years, advancements in applications of gene therapy have accelerated. Gene therapy aims to treat and potentially cure people with genetic diseases that have a substantial effect on quality of life and for which no or few therapeutic options are available. As developments continue at a rapid pace, promising outcomes frequently feature prominently in the news. In November, 2023, the UK Medicines and Healthcare products Regulatory Agency gave the first conditional market authorisation of a CRISPR-based genome-editing therapy for the treatment of people with sickle cell disease and transfusion-dependent β-thalassaemia (TDT) for whom haematopoietic stem cell transplantation is appropriate but not available. The US Food and Drug Administration and the European Commission followed shortly thereafter. In the UK and EU, an estimated 10,000 people with sickle cell disease and TDT are potentially eligible for treatment.

These approvals were delivered after encouraging interim efficacy results from one clinical trial for each disease. The results, published in The New England Journal of Medicine , revealed that most people who received treatment remained free from severe vaso-occlusive crises in sickle cell disease or did not need red blood cell transfusion in TDT for at least 12 consecutive months. Sickle cell disease and TDT affect the ability of red blood cells to carry oxygen and are caused by genetic mutations in both copies of the HBB gene encoding haemoglobin. Rather than editing each mutation of individual people, the strategy of this treatment approach (known as exagamglogene autotemcel [exa-cel]) is to reinstate the expression of fetal globin genes , which are postnatally silenced by transcription factors such as BCL11A. More specifically, the enhancer regulating the expression of BCL11A is inactivated by ex-vivo genome-editing in bone-marrow stem cells, allowing them to express fetal haemoglobin. The cells are then transplanted back, where they can proliferate in the bone marrow.

In a study published in The New England Journal of Medicine in January, 2024, Longhurst and colleagues also used an indirect strategy for hereditary angioedema, thanks to which eligible participants could be administrated the same in-vivo CRISPR-based gene-editing therapy. Instead of editing the mutations in SERPING1 , the gene causing the disease, the gene-editing strategy targeted the KLKB1 gene encoding plasma kallikrein, a downstream protease in the dysregulated contact activation pathway. Ten participants received a single dose of the treatment. No severe adverse events were detected, plasma kallikrein concentrations were reduced for at least 24 weeks, and exploratory analyses promisingly showed a reduction in the number of angioedema attacks per month.

As discussed in a Review published in The Lancet in February, 2024, a core advantage of gene or genome-editing therapies over gene-addition therapies is its potential to expand the spectrum of targetable diseases to autosomal dominant genetic disorders, for which adding a non-mutated gene is not an adequate solution. Instead, genome-editing strategies can directly repair disease-causing mutations or modify gene or genome sequences. They also overcome a limitation of gene-addition therapies whereby the expression of the added gene, typically controlled by promoters and enhancers different from those found in nuclei of human cells, have little fine-tuning. Reflecting its versatility, therapeutic applications and ongoing clinical trials based on genome-editing strategies cited in this Review are as diverse as cancer immunotherapy, HIV infection, type 1 diabetes, or organ xenotransplantation.

Yet, the risk of unwanted effects exists and is linked to off-target editing (ie, unintended genetic modifications) and immunogenicity against the engineered nucleases, such as Cas9, or the viral-based vectors delivering the molecular components of this technology. Basic research and preclinical studies continue to provide computational and biological tools that help reduce this risk. For example, in a study published in Molecular Therapy in August, 2023, Stahl and colleagues optimised the transient delivery of Cas9 ribonucleoproteins in the mouse brain, leading to reduced immune responses but similar editing capacity in neurons to a commonly used adeno-associated virus (AAV)-based delivery.

Gene-addition therapies are complementary approaches to genome editing and, despite being used to treat human diseases for more than a decade, clinical trials continue to test their breadth of therapeutic applications. In a single-arm, single-centre trial published in The Lancet in May, 2024, Lv and colleagues used a gene-addition strategy to treat children who, due to biallelic mutations in the OTOF gene, had autosomal recessive deafness 9. A single injection of an AAV carrying a human OTOF transgene into the cochlea of six children aged 1–6 years with severe-to-complete hearing loss resulted in no dose-limiting toxicity or serious adverse events; five children had hearing recovery and improved speech perception.

However, successes in gene therapies should not make us forget disappointing outcomes. In June, 2024, Pfizer announced that boys aged 4–7 years with Duchenne muscular dystrophy treated with fordadistrogene movaparvovec , an AAV carrying a shortened version of the dystrophin gene, had no improvement of motor function 1 year after treatment compared with placebo. Besides no efficacy, safety concerns can also be a reason for poor outcomes. The ASPIRO trial evaluating the safety and efficacy of a single intravenous infusion of resamirigene bilparvovec was stopped after the death of four participants. The investigational AAV-based gene-replacement therapy of MTM1 was administered to boys younger than 5 years who had X-linked myotubular myopathy (XLMTM) and required long-term mechanical ventilation. Exploratory analyses of the trial reported in The Lancet Neurology highlighted that undiagnosed cholestatic liver disease in children with XLMTM can potentially interact with the gene therapy, leading to serious hepatic and hepatobiliary adverse events and progressive liver disease unresponsive to immune suppression. Nevertheless, a promising reduction in ventilator dependence and improvement of motor function were observed in some surviving participants. In a companion Article published in eBioMedicine , Lawlor and colleagues reported a histopathological analysis of muscle biopsies showing improvement of organelle localisation and myofiber size 24 weeks after treatment.

Dowling and colleagues published in Nature Medicine in July, 2024, a study in which a single participant aged 4 years with hereditary spastic paraplegia type 50 was administrated an AAV-based gene-addition therapy of the target gene, AP4M1, intrathecally. After 12 months, no serious adverse events were detected and preliminary outcomes suggested that the progressive disease course, including developmental delay, microcephaly, and loss of motor skills, might be inhibited. Dowling and colleagues discussed time and costs as key aspects to consider when developing a single-gene therapeutic. In this case, development took almost 3 years from diagnosis to dosing and cost more than CA$3,500,000.

Affordability of treatment for all individuals in need remains an unsolved challenge in gene therapy. Although more than 75% of people with sickle cell disease are born in Africa, as reminded by Munung and colleagues in their review published in Gene Therapy , exa-cel trials have not involved any African countries so far. More generally, Munung and colleagues identified ethical, legal, and social issues that might need to be overcome for the implementation of gene therapy for people with sickle cell disease in Africa. Moreover, the scarce availability of genetic testing in many low-income and middle-income countries and the under-representation of diverse ethnic populations in genomic datasets and genetic research, impeding our understanding of genetic variants in many populations, are initial barriers keeping many individuals far from gene therapy.

As gene therapy enters the area of genome editing, a multidisciplinary and collaborative research effort is required to provide an efficient, safe, and affordable solution for all individuals with burdensome genetic disorders. In this field, basic and preclinical research have made and continue to make invaluable contributions to the clinic by deciphering biological underpinnings of diseases and providing the therapeutic tools to treat them. eBioMedicine welcomes translational research that addresses some of the challenges in this fundamentally different medical approach, in which a cure rather than a treatment might be achievable.