Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Diamond-Blackfan anemia (DBA) is a rare, life-threatening genetic blood disorder. It impairs the bone marrow's ability to ...

Several years of preliminary research results suggest chimeric antigen receptor (CAR) T-cell therapy could benefit pediatric patients with refractory systemic lupus erythematosus (SLE). The gene ...

Cardiovascular disease remains the foremost cause of morbidity and mortality globally, affecting millions of individuals. Recent discoveries illuminate the substantial role of genetics in ...

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...

The global cell and gene therapy market size was valued at USD 30.27 billion in 2024 and is projected to reach USD 190.82 ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

Clinical Trial Success at Cincinnati Children's Supports Recent US FDA Approval for First Therapy to Directly Replace a Gene in the Brain ...

AAV is the top vector choice for use in gene therapies for its stable expression, strong biosafety rating, low immunogenicity, convenient ocular delivery routes and high product stability. Dr. Lu will ...