Gene therapy is a promising therapeutic procedure for genetic disorders or diseases in which defective genes are corrected, replaced, or inactivated.
In the case of adrenoleukodystrophy (ALD) a genetic disorder caused by mutations in the ABCD1 gene that damages the myelin sheath around nerve cells gene therapy may benefit patients prior to the onset, or during the early stages, of the disease by stopping the progression of demyelination. However, the therapy cannot be beneficial after the disease has worsened significantly.
Gene therapy works by introducing the correct gene sequence into cells. Since genetic material cannot enter the cell on its own, the correct gene sequence needs to be delivered using a vector. This vector can be a modified virus that has been engineered to remove its pathogenic genetic material so that it cannot cause disease, but is still able to transfer the correct gene sequence to the host cell.
The vector can be directly injected into the patients body or into host cells grown in the laboratory and then transplanted back into the patient. Upon successful viral transfer, the host cell should be able to produce the functional protein.
In ALD, the clinician first takes out the patients own stem cells (autologous) and then inserts the correctABCD1 gene sequence into these cells using a viral vector in the laboratory. The corrected stem cells that are able to produce the functional ALD protein are then implanted back into the patients body so they may develop into nerve cells in the brain. Since the patients own cells are being used, there are fewer risks than when donor stem cells are used.
Lenti-D,an investigational gene therapy developed by Bluebird Biois currently being studied in a Phase 2/3 clinical trial (NCT01896102) in the U.S., the U.K., and France. The study aims to evaluate the safety and effectiveness of Lenti-D in boys, up to 17 years old who havecerebral adrenoleukodystrophy (CALD). Based on the preliminary data from this study,the U.S. Food and Drug Administration (FDA)designated Lenti-D a breakthrough therapy for the treatment of CALD in May 2018.
A Phase 1/2 clinical trial (NCT02559830) is recruiting patients with ALD at the Shenzhen Second Peoples Hospital in Guangdong, China. The study aims to assess the safety and effectiveness of transplanting patient-derived bone marrow stem cells, which have been genetically-corrected using a lentiviral vector, for the treatment of ALD.
Another Phase 1/2 clinical trial (NCT03727555) at the Shenzhen Geno-Immune Medical Institute also in Guangdong, China is recruiting 10 patients with ALD. The study aims to evaluate the safety and effectiveness of a lentiviral vector carrying the healthy ABCD1 gene (TYF-ABCD1) injected directly into the patients brain for the treatment of ALD.
***
Adrenoleukodystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
Sorry! The Author has not filled his profile.
Total Posts: 0
zge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
Continued here:
Gene Therapy - Adrenoleukodystrophy News
- Patient Dies of Acute Liver Failure After Treatment With Sareptas DMD Gene Therapy Elevidys - CGTLive - March 19th, 2025
- Patient dies following muscular dystrophy gene therapy, Sarepta reports - The Associated Press - March 19th, 2025
- Duchenne patient dies after receiving Sarepta gene therapy - March 19th, 2025
- Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys - MedCity News - March 19th, 2025
- DoD grant funds Hollings researcher's idea to pursue gene therapy for cancer - Medical University of South Carolina - March 19th, 2025
- Recon: Sarepta reports death of teen who received Duchenne gene therapy; Novartis to slash 427 jobs while revamping cardiovascular business -... - March 19th, 2025
- Data Gaps Leave Long-Term Impact of Ex Vivo Gene Therapy in DMD Uncertain - AJMC.com Managed Markets Network - March 19th, 2025
- CHO Plus Obtains U.S. Patent for Improved Production of Viral Vectors for Gene Therapy - Business Wire - March 19th, 2025
- Sarepta Shares Fall on Report of Patient Death After Gene Therapy - Bloomberg - March 19th, 2025
- Hologen AI commits up to $430M to help take MeiraGTx's Parkinson's gene therapy through phase 3 and beyond - Fierce Biotech - March 19th, 2025
- Duchenne patient on Sareptas gene therapy dies - The Business Journals - March 19th, 2025
- Im Unstoppable: New gene therapy cures first New Yorker of sickle cell anemia - PIX11 New York News - March 19th, 2025
- Boost in cancer treatment: PGI working on lab for stem cell, gene therapies - The Times of India - March 19th, 2025
- Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy - Forbes - March 19th, 2025
- Sarepta says teen died after its gene therapy treatment By Reuters - Investing.com - March 19th, 2025
- Innovative Gene Therapy Approach Drives Buy Rating for Insmed in DMD Treatment - TipRanks - March 19th, 2025
- Sarepta says patient dies after treatment with gene therapy - TradingView - March 19th, 2025
- Sarepta tumbles after patient dies following gene therapy treatment - TradingView - March 19th, 2025
- MeiraGTx teams with cryptic AI startup, co-founded by Eric Schmidt, to advance Parkinson's gene therapy - Endpoints News - March 19th, 2025
- Sickle cell anemia patient reunites with Long Island doctors whose gene therapy treatments made him symptom-free - Newsday - March 19th, 2025
- Extracellular vesicles for the delivery of gene therapy - Nature.com - March 9th, 2025
- Around the Helix: Cell and Gene Therapy Company Updates March 5, 2025 - CGTLive - March 9th, 2025
- Inside the secret island where wealthy people go to alter their DNA - Daily Mail - March 9th, 2025
- Regenerons Gene Therapy DB-OTO Trial Shows Promising Hearing Improvement - The Hearing Review - March 9th, 2025
- Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032 | DelveInsight - GlobeNewswire - March 9th, 2025
- College Station gene therapy company partners with nonprofit to develop treatments for rare diseases - KBTX - March 9th, 2025
- World Hearing Day 2025: Looking Back at Progress in Gene Therapy - CGTLive - March 9th, 2025
- Reflecting on a milestone year for cell and gene therapies - Pharmaceutical Technology - March 9th, 2025
- Q&A: Whats Next for Hemophilia Gene Therapy? | Newswise - Newswise - March 9th, 2025
- 'Llife-changing' gene therapy in London partially restores CT child's sight - CT Insider - March 9th, 2025
- The Genesis of Cell Therapy: Bridging Traditional Pharmacology and Gene Therapy - Technology Networks - March 9th, 2025
- Regenxbio at TD Cowen Conference: Gene Therapy Advancements - Investing.com - March 9th, 2025
- Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas - Business Wire - March 9th, 2025
- Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS... - March 9th, 2025
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024