Health ❯ Healthcare ❯ Genetics

Gene Therapy

Rare Diseases Beta Thalassaemia Gene Editing Individualized Treatment GBA1 Gene Variants Personalized Medicine Immune Diseases Antisense Oligonucleotides ADA-SCID CRISPR Technology

3 ARTICLES

last mo.

Louisiana Man Declared Functionally Cured of Sickle Cell After Gene‑Editing Therapy

Doctors say edited versions of his own stem cells have halted disease activity, a result that signals medical promise while revealing the treatment’s complexity, cost and limited availability

3 ARTICLES

9mo ago

Scientists Identify ZBTB16-Driven T-Cell Subtype Behind Treatment-Resistant Childhood Leukemia

11 ARTICLES

9mo ago

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

5 ARTICLES

last yr.

Ambroxol Trial Confirms Safety, Brain Delivery and Early Cognitive Benefits in Parkinson’s Dementia

3 ARTICLES

last yr.

First In Vivo CRISPR Gene Therapy Saves Newborn with Rare Genetic Disorder

6 ARTICLES

last yr.

Philadelphia Doctors Pioneer Personalized Gene Therapy for Rare Metabolic Disorder

4 ARTICLES

last yr.

UCSF Research Advances Prenatal Therapy for Spinal Muscular Atrophy

6 ARTICLES

last yr.

Gene Therapy Restores Immune Function in Children with Rare Disorder

6 ARTICLES

2y ago

NHS Approves Groundbreaking Gene-Editing Therapy for Beta Thalassemia