I have heard about people using genes to treat diseases nowadays, but I am not sure what this gene therapy means.
Gene therapy involves trying to alter or modify the genes inside your bodys cells in order to treat or stop a disease.
Since 2017, the US Food and Drug Administration (FDA) has approved three different types of gene therapy.
Maybe we can start at the beginning: what are genes?
Genes are the basic physical and functional unit of heredity.
Our genes are made out of DNA (deoxyribonucleic acid).
Each person has two copies of each gene one inherited from your mother and the other inherited from your father.
Each human being has around 20,000 to 25,000 genes.
These genes code for the way your body and mind are structured.
Some genes act as instructions (a blueprint) for your body to make various proteins, which in turn form your cells and organs, and the enzymes and hormones that regulate your body.
Other genes do not code for proteins.
Most genes are the same for all human beings, which is why we all look like human beings (and not a kangaroo, fish, bird or an alien)!
However, just under 1% of our genes vary slightly between each person.
That is why we have different races, heights, propensity for different diseases, curly or straight hair, etc.
These small differences also contribute to why we all look different from one another.
Genes that dont work as they should also cause diseases in the human body.
What types of diseases are caused by faulty genes?
These are what we call genetic disorders.
A genetic disease is any type of disease caused by an abnormality in our genetic blueprint.
This abnormality can range from very minor to significantly major.
What we consider minor is, for example, a small mutation in the DNA of a single gene resulting in the change of a single base protein.
What we consider major is a gross chromosomal abnormality, such as the addition of a whole chromosome or the subtraction of one.
Some genetic disorders are inherited from our parents.
Others are caused by mutations due to our environment.
Examples of single gene disorders, which are caused by the alteration of just one gene in our bodies, are:
Examples of multifactorial inheritance, which are caused by a combination of environmental factors and mutations in many of our genes, are:
If we inherited these genes from our parents, then how can we possibly modify or alter them? This sounds terribly like science fiction.
We are rapidly approaching that era where what used to be science fiction could become part of our everyday life.
In gene therapy, scientists can:
How do they do this? Do they have to harvest my cells? Im scared just thinking about it!
Many of the vectors are viruses, especially adenoviruses (not coronaviruses!).
Viruses have a natural ability to deliver genetic material into our cells.
After all, their main purpose is to attach themselves to cells and reproduce themselves.
Sometimes, the vector or virus is injected straight into our bodies, where they will deliver the gene that will modify our cells.
They are injected straight into the part of our body that has those defective cells.
Other times, we have to harvest healthy tissue from our body that needs to be modified.
These are usually tissues containing immune cells or stem cells, e.g. blood or bone marrow.
These tissue samples are then taken to the lab and specific cells are separated out.
The viral vector containing the corrective gene is then introduced to the harvested cells in the lab.
The modified cells are left to multiply, and then injected back into us.
Once inside our bodies, they will continue to multiply and eventually treat the disease or correct the defect within us.
Learn more about gene therapy in the next Tell Me About column on Dec 31 (2020).
Dr YLM graduated as a medical doctor, and has been writing for many years on various subjects such as medicine, health, computers and entertainment. For further information, email email@example.com. The information contained in this column is for general educational purposes only. Neither The Star nor the author gives any warranty on accuracy, completeness, functionality, usefulness or other assurances as to such information. The Star and the author disclaim all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.
Read more from the original source:
- Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT - February 17th, 2021
- Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and... - February 17th, 2021
- Avance Biosciences Expanding Houston Campus in Support of Cell and Gene Therapy Drug Development - BioSpace - February 17th, 2021
- Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com - February 17th, 2021
- Gene Therapy GS010 Safe, Well-Tolerated for LHON Patients - MD Magazine - February 17th, 2021
- bluebird bio 'baffled' after NICE rejects beta-thalassaemia gene therapy - - pharmaphorum - February 17th, 2021
- Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of... - February 17th, 2021
- GenSight Biologics' gene therapy proves safe in LHON trial - Clinical Trials Arena - February 17th, 2021
- Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference - GlobeNewswire - February 17th, 2021
- Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive - February 17th, 2021
- Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe... - February 17th, 2021
- CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way - FiercePharma - February 17th, 2021
- Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board - Business Wire - February 17th, 2021
- bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes - February 17th, 2021
- Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -... - February 17th, 2021
- Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -... - February 17th, 2021
- Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace - February 17th, 2021
- Gene Therapy Market to Reflect Impressive Growth Rate During 2020-2027 AveXis, Vineti, uniQure NV, Spark Therapeutics KSU | The Sentinel Newspaper -... - February 17th, 2021
- Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence - BioSpace - February 17th, 2021
- Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases - BioSpace - February 11th, 2021
- Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health... - February 11th, 2021
- The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire - February 11th, 2021
- FLT201 Shows Promise as a Gene Therapy for Gaucher Disease - MD Magazine - February 11th, 2021
- NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bio's Gene Therapy for Life-Limiting Rare Blood Diseas - PharmiWeb.com - February 11th, 2021
- Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of... - February 11th, 2021
- Breakthrough gene therapy helps Utah boy thrive - Yahoo News - February 11th, 2021
- Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance - February 11th, 2021
- Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo... - February 11th, 2021
- Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire - February 11th, 2021
- Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma - February 11th, 2021
- FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare... - February 11th, 2021
- Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key... - February 11th, 2021
- FDA approves third gene therapy for large B-cell lymphoma - European Pharmaceutical Review - February 11th, 2021
- Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire - February 7th, 2021
- FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra - February 7th, 2021
- Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace - February 7th, 2021
- The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -... - February 7th, 2021
- AGTC Executives Awarded First Place in the BioProcess International Reader's Choice Awards, Cell & Gene Therapies Category - GlobeNewswire - February 7th, 2021
- Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace - February 7th, 2021
- Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace - February 7th, 2021
- Welsh, Carson, Anderson & Stowe Commits $250 Million in a Strategic Partnership with Kiniciti, a Newly-Formed Platform Investing in Cell and Gene... - February 7th, 2021
- Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)... - February 7th, 2021
- Abeona Therapeutics Announces Clinical Investigator Webinar to Review ABO-102 and ABO-101 Clinical Data Presented at the 17th Annual WORLDSymposium -... - February 7th, 2021
- Decibel and Catalent Sign Development and Manufacturing Agreement for Dual-Vector Gene Therapy for the Treatment of Congenital Hearing Loss - Yahoo... - February 7th, 2021
- Meeting the commercialization challenge of a surging gene and cell therapy market - FierceBiotech - February 7th, 2021
- Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com - February 7th, 2021
- Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - BioSpace - February 7th, 2021
- Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly - Business Weekly - February 7th, 2021
- Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook - Business Wire - February 7th, 2021
- FDA's New Guidance on Cell and Gene Therapy Recommends Assessment of COVID-19 Transmission Risks - Lexology - February 7th, 2021
- After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News - December 17th, 2020
- Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist - December 17th, 2020
- After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News - December 17th, 2020
- Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,... - December 17th, 2020
- Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire - December 17th, 2020
- Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma - December 17th, 2020
- Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -... - December 17th, 2020
- Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes - December 17th, 2020
- They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News - December 17th, 2020
- Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire - December 17th, 2020
- Gene Therapy Injection in One Eye Improves Vision in Both - Technology Networks - December 17th, 2020
- Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace - December 17th, 2020
- Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -... - December 17th, 2020
- Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire - December 17th, 2020
- Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace - December 17th, 2020
- Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas - December 17th, 2020
- Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage - December 17th, 2020
- Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire - December 14th, 2020
- ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech - December 14th, 2020
- Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -... - December 14th, 2020
- Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com - December 14th, 2020
- Gene Therapy, Absolutely and For Real | In the Pipeline - Science Magazine - December 14th, 2020
- Treatment with Investigational LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111) Results in Complete Elimination of SCD-Related Severe... - December 14th, 2020
- Rocket Pharmaceuticals in orbit after gene therapy read-out - - pharmaphorum - December 14th, 2020
- Bluebird trumpets long-term data from beta-thalassaemia gene therapy - - pharmaphorum - December 14th, 2020
- ASH 2020: Novel Gene Therapy Found to be Safe and Effective in Treatment of Hemophilia B - OncoZine - December 14th, 2020
- Gene Therapy Unexpectedly Improves Vision In Both Eyes Of Patients Suffering A Form Of Blindness - IFLScience - December 14th, 2020
- Innovative payment models to support cell and gene therapies on the rise - MedCity News - December 14th, 2020
- Better education needed to give patients improved understanding of gene therapies, new review highlights - University of Birmingham - December 14th, 2020
- Global Gene Therapy Market Report 2020: Market is Expected to Recover and Reach $6.84 Billion in 2023 - Forecast to 2030 - GlobeNewswire - December 14th, 2020