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

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 ...

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

Deficiency in adenosine deaminase (ADA) was the first SCID condition for which a genetic and molecular cause was identified 1.The absence of ADA leads to the accumulation of (de)adenosine ...