BioSpace7 小时
RNA Editing Hits the Clinic, Fueling New Hope for Rare and Common Diseases
With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery ...
Pharmaceutical Technology18 天
Italian charity seeks EMA approval for rescued gene therapy
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 ...
Italian charity seeks EMA approval for rescued gene therapy
Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.
Nature19 天
20 years of gene therapy for SCID
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 ...
The Lancet23 天
Rising mpox trends in DR Congo: the neglected spread of an epidemic
bSkin Neglected Tropical Diseases and Sexually Transmitted Infections Section, Fight Infectious Diseases Foundation, University Hospital Germans Trias i Pujol, 08917 Badalona, Spain ...
Frontiers2 年
Secondary myoadenylate deaminase deficiency is not a common feature of inflammatory ...
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 ...
Frontiers3 年
Plasma Adenosine Deaminase (ADA)-1 and -2 Demonstrate Robust Ontogeny Across the First Four ...
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) ...