ADA-SCID


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Related to ADA-SCID: Adenosine deaminase deficiency
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Words related to ADA-SCID

SCID resulting from mutation of a gene that codes for adenosine deaminase

References in periodicals archive ?
Revcovi is a PEGylated recombinant adenosine deaminase enzyme developed by Leadiant Biosciences to treat ADA-SCID.
ADA-SCID is an ultra-rare, inherited genetic disorder, caused by a deficiency in the ADA enzyme that is fatal if left untreated.
In ADA-SCID, ability to metabolise adenosine leads to rectification of the immune deficit (20).
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.
Only five previous patients with ada-SCID have been successfully treated in the same way, all in Milan, Italy.
The company is developing the gene therapy in collaboration with University College London (UCL), Great Ormond Street Hospital (GOSH), and the University of California, Los Angeles (UCLA), as a treatment for adenosine deaminase severe combined immunodeficiency (ADA-SCID) - a rare inherited disorder of the immune system.
Therapeutic areas included ZFN-based approaches to infectious diseases such as HIV/AIDS and monogenic diseases such as hemophilia, ADA-SCID, retinal neurodegeneration and epidermolysis bullosa.
The clinical data we've seen to date with Orchard's therapy for ADA-SCID is exceptional, and the company has assembled and impressive R&D pipeline to apply the approach more widely.
It was reported on Friday that the grant will fund a new clinical trial for Severe Combined Immunodeficiency caused by adenosine deaminase deficiency, commonly known as ADA-SCID or "bubble baby" disease.
The European Commission has approved Strimvelis, an ex-vivo stem cell gene therapy intended for the treatment of a very rare disorder called ADA-SCID, or severe combined immunodeficiency due to adenosine deaminase deficiency, it was reported yesterday.
Biotechnology company MolMed S.p.A (Milan: MLM.MI) announced today the signing of an agreement with GlaxoSmithKline (GSK)(LSE:GSK) under which MolMed will develop a production process for an investigational gene therapy for ADA-SCID (Adenosine Deaminase Deficiency - Severe Combined Immune Deficiency).
5 August 2011 - Italy-based biotechnology company Molecular Medicine SpA (MolMed) (BIT:MLM) announced today that it has entered into an agreement with UK-based healthcare group GlaxoSmithKline (LON: GSK), under which MolMed will develop a production process for an investigational gene therapy for ADA-SCID (Adenosine Deaminase Deficiency - Severe Combined Immune Deficiency).
Under the agreement, GSK will gain an exclusive licence to develop and commercialise an investigational gene therapy, for ADA Severe Combined Immune Deficiency (ADA-SCID) - a rare and life-threatening immune deficiency, which affects approximately 350 children worldwide.