Over the past 15 years, breakthroughs in basic science have revealed the genetic etiology for several inherited cardiovascular diseases. [1,2] By studying instructive monogenic diseases ...

Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease community.

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions ...

Dyno Therapeutics has recently announced the formation of a new strategic partnership with Swiss pharma giant Roche to ...

With the formation of a new strategic partnership, Dyno Therapeutics is responsible for the development of novel AAV capsids.

Researchers at Stanford Medicine have developed an AI-based method to identify complex structural variants in the human ...

Cantor Fitzgerald analyst Kristen Kluska maintained a Buy rating on Taysha Gene Therapies (TSHA – Research Report) yesterday and set a ...

In this case report, Gad and colleagues performed corneal confocal microscopy to assess for evidence of nerve regeneration ...

NIH study highlights potential for earlier health interventions in individuals of West African ancestry. Researchers from the ...

A study by researchers at the National Institutes of Health (NIH) and their collaborators has revealed a significant genetic ...

London – 28 October 2024 - Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, presented preclinical data at the American Society of ...

The findings ... Aug. 29, 2024 — For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene ...