The Conversation

Gene therapy can be less effective in women – and my research in mice brings us one step closer to understanding why

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...
Forbes

Gene Editing Or Gene Silencing: The Therapies Poised To Transform Medicine

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
The American Journal of Managed Care

Progress and Challenges With Gene Therapy in Hereditary Hearing Loss

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...
Morningstar

Fondazione Telethon and Orphan Therapeutics Accelerator Sign Memorandum of Understanding to Pioneer Non-Profit Commercial Access Model for Ultra-Rare Disease Gene Therapy in the US

Collaboration Aims to Establish Sustainable Access to a Newly Approved Therapy Targeting a Rare Immunodeficiency, Filling Urgent Gap Left by For-Profit Efforts CAMBRIDGE, Mass. and ROME, Dec. 15, 2025 ...
TCTMD

CRISPR-Cas9 Gene-Editing Therapy Shows Early Promise for Dyslipidemia

NEW ORLEANS, LA—An investigational CRISPR-Cas9 gene-editing therapy that targets angiopoietin-like protein 3 (ANGPTL3), which has a role in regulating lipid metabolism, appears to safely lower levels ...
Medical Xpress

X-SCID gene therapy gives scientists a rare glimpse into early immune system

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. The work stems ...
The Scientist

CRISPR Therapy Progress and Prospects

Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins are core components of fast-evolving therapeutic gene editing tools. Scientists have used CRISPR ...
Drexel University

Breakthrough on Gene Therapy for Hereditary Spastic Paraplegia

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College of Medicine and the UMass Chan Medical School have achieved ...

Rare cranial disorders: Towards a non-invasive therapy using gene silencing delivered by nanoparticles and 3D printing

A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...

Beyond Opioids: The New Gene Therapy That Relieves Pain Without the Addiction

The new method is designed to focus specifically on pain-related signals, without interfering with normal activity in other parts of the brain. A new preclinical study has identified a gene therapy ap ...
Hackaday

Gene Therapy Aims To Slow Huntington’s Disease To A Crawl

Despite the best efforts of modern medicine, Huntington’s disease is a condition that still comes with a tragic prognosis. Primarily an inherited disease, its main symptoms concern degeneration of the ...

Cell and Gene Therapy Investment Trends Strategic Intelligence Report 2025: Market to Reach $63 Billion by 2031 - ResearchAndMarkets.com

The market size of CGT products is expected to continue to rise by 2031 and reach $63 billion. CGTs have yielded commercial gains for several pharma companies. For example, some of the top-selling CGT ...