Gene Therapy News and Research

RSS

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

How processed red meat might drive neurodegenerative diseases

The relationship between processed red meat and neurodegenerative diseases is complex, with potential risks and protective effects requiring more investigation.

15 Sep 2025

CRISPR-based therapeutic approach designed to treat fatal pediatric disease

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood.

11 Sep 2025

Abselion launches AAVX and AAV9 Total Capsid Quantification Kits

Abselion, a pioneering life sciences technology company focused on simplifying biomolecule quantification, has expanded its product offering, with the launch of the AAVX Total Capsid Quantification Kit and the AAV9 Total Capsid Quantification Kit.

9 Sep 2025

Prefrontal cortex biopsies during deep brain stimulation surgery do not increase risk of adverse events

Obtaining prefrontal cortex biopsies during deep brain stimulation (DBS) surgery in living patients does not increase the risk of adverse events or cognitive decline compared to standard DBS procedures that don't involve biopsies, a team of clinical research scientists at the Icahn School of Medicine at Mount Sinai has demonstrated.

4 Sep 2025

Atelerix and Rodon Global partner to integrate cryo-free preservation technology into specialist logistics solutions

Atelerix, a biotech company revolutionizing cell preservation and biological transport with its pioneering hydrogel encapsulation technology, today announced an agreement with Rodon Global (“Rodon”), the established freight company focused on clinical trials and drug discovery headquartered in South Africa.

4 Sep 2025

Breakthrough reveals how viruses efficiently pack genetic material

Researchers at San Diego State University and Michigan State University are shedding new light on how viruses meticulously pack their genetic material - a breakthrough that could help researchers engineer antivirals and gene therapies.

2 Sep 2025

Clonogenic hepatocytes identified as engines of liver development

In a new study published in the Journal of Hepatology, researchers from the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) show that only 15–20% of hepatocytes in newborn mice - dubbed clonogenic hepatocytes - are responsible for generating over 90% of the adult liver mass.

29 Aug 2025

Gene editing therapy exa-cel improves quality of life in sickle cell disease and beta thalassemia

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two studies published in Blood Advances.

27 Aug 2025

How leadership drives inspection readiness from the top down

Discover how proactive leadership sets the tone for inspection readiness across all levels of an organization.

From DSI, a PLG Company 25 Aug 2025

Strategic tips for early-stage animal health companies facing regulatory challenges

Karen Greenwood shares practical advice for early-stage animal health companies navigating FDA CVM processes.

From DSI, a PLG Company 25 Aug 2025

ChronODE method offers precision in timing gene therapy treatments

A Yale research team has created a new computer tool that can pinpoint when exactly genes turn on and off over time during brain development - a finding that may one day help doctors identify the optimal window to deploy gene therapy treatments.

20 Aug 2025

Texas Children’s successfully treats AADC deficiency using FDA-approved gene therapy

Texas Children's is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency.

19 Aug 2025

Improving gene therapy safety with human kidney organoids

Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids."

19 Aug 2025

Dual vector gene therapy for GM2 gangliosidosis shows encouraging results in clinical trial

Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions.

18 Aug 2025

Advancing cancer treatment with targeted microrobotic swarms

Cancer remains one of the most challenging diseases to treat, as conventional therapies like chemotherapy and radiotherapy are often hindered by poor specificity, systemic toxicity, and damage to healthy tissues.

18 Aug 2025

New AAV gene therapy reverses age-related macular degeneration

Neovascular age-related macular degeneration (nAMD) is a progressive eye disease characterized by choroidal neovascularization and subretinal hemorrhage and exudation, leading to vision impairment. Current first-line treatment for nAMD are anti-VEGF agents, which has shown promising therapeutics outcomes.

18 Aug 2025

New generation of CRISPR shows safer path to treating genetic diseases

A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases like Sickle Cell, while also proving beyond doubt that chemical tags on DNA - often thought to be little more than genetic cobwebs - actively silence genes.

17 Aug 2025

Gene editing restores hearing and balance in adult mice with DFNA41 deafness

Zheng-Yi Chen, DPhil, associate scientist at the Eaton-Peabody Laboratories, and Ines and Fredrick Yeatts Chair in Otolaryngology, at Mass Eye and Ear, is the senior and co-corresponding author of a paper published in the Journal of Clinical Investigation, "Single Dose Genome Editing Therapy Rescues Auditory and Vestibular Functions in Adult Mice with DFNA41 Deafness."

14 Aug 2025

New method greatly improves the precision of genome editing

A research team headed by the University of Zurich has developed a powerful new method to precisely edit DNA by combining cutting-edge genetic engineering with artificial intelligence.

12 Aug 2025

Prime editing reverses symptoms of severe childhood neurological disease

Scientists use a precise form of gene editing called prime editing to correct the most common genetic mutations that cause alternating hemiplegia of childhood, a rare and severe neurological disorder that begins in infancy.

From The Jackson Labs 6 Aug 2025