With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...

The clinical features of MAS overlap with those of active sJIA, complicating early diagnosis and treatment. We evaluated adenosine deaminase 2 (ADA2), a protein of unknown function released ...

An Italian foundation that helped a young boy overcome Wiskott-Aldrich syndrome becomes the first non-profit in the world to ...

Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

After hours: February 21 at 5:37:37 PM EST Loading Chart for ROIV ...

It previously did so with simoladagene autotemcel, a therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCiD). That gene therapy also originated in the laboratories of the San ...

RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or ...