Gene therapy restores key fragile X traits in preclinical study

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant ...
FierceBiotech

Beam gene therapy for protein deficiency passes early safety test, sets efficacy bar in space: analyst

Boston biotech Beam Therapeutics has announced initial data from a phase 1/2 trial of its gene therapy for a genetic lung and liver disease, with no serious adverse events reported among the nine ...
CNN

Experimental gene therapy found to slow Huntington’s disease progression, company says

An experimental gene therapy was found to slow the progression of Huntington’s disease in a “pivotal” Phase 1/2 study, according to drugmaker uniQure, marking a major step toward a potential first ...
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 ...
Labroots

Optimizing cell and gene therapy with serological pipetting

Streamlining cell and gene therapy workflows with precise serological pipetting Cell and gene therapies are transforming the ...
Medscape

The Gene Therapy Poised to Rewrite Childhood Deafness

Four decades have passed since cochlear implants gave infants born deaf the ability to hear. Now, gene therapy promises to restore natural hearing for those born with a rare form of deafness, and the ...
Medical Xpress

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to a unique genetic mutation to save an infant named Baby KJ from dying of a ...
Forbes

Genetic Swiss Army Knife: New Tool For Gene Editing And Therapy

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...
Saint Louis University

Ethical, Legal and Social Implications: Bringing Gene Therapies to the Clinic

Vence L. Bonham Jr., Acting Deputy Director at the National Human Genome Research Institute (NHGRI) and an investigator in the National Human Genome Research Institute’s (NHGRI) Division of Intramural ...