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 ...

Background: Myoadenylate deaminase (MAD) deficiency is a form of metabolic myopathy, which generally causes only mild symptoms in the primary inherited form. Inflammatory myopathies are a group of ...

We thank Kristin Johnson of Boston Children’s Hospital for artwork in Figure 1. ADA, adenosine deaminase; cGAS, cyclic guanosine monophosphate–adenosine monophosphate; CXCL, chemokine (C-X-C motif) ...