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Samples in periodicals archive:
Sustained Correction of X-Linked Severe Combined Immunodeficiency by Ex Vivo Gene Therapy," New England Journal of Medicine 346 (2002): 1185-93; A.
Children with X-linked severe combined immunodeficiency, a rare condition that affects only males and is sometimes referred to as "baby in the bubble syndrome", die within their first year without treatment.
A US government health panel has recommended that 2 US gene therapy trials involving children with X-linked severe combined immunodeficiency (X-SCID) be allowed to resume but only on a limited basis.
National Institutes of Health and the Hopital Necker in Paris, France, have separately announced success in using this technique to create effective gene therapies for chronic granulomatous disease, a rare congenital blood disorder, and X-linked severe combined immunodeficiency (SCID), an inherited immune disorder, respectively.
Sangamo is also developing sequence-specific ZFP Nucleases (ZFNs) for therapeutic gene modification as a treatment for a variety of monogenic diseases such as X-linked severe combined immunodeficiency (X-linked SCID) and sickle cell anemia (SCA) and infectious diseases such as HIV.
In a male fetus with X-linked severe combined immunodeficiency (SCID), in utero transplantation of cryopreserved fetal liver cells led to complete T and NK cell reconstitution, according to a report from researchers at Huddinge University Hospital in Sweden.
NASDAQ:NEXL), one of the world's leading suppliers of therapeutic and diagnostic products based on stem cell technology, announced today that French researchers used the Company's Lifecell(R) X-Fold(TM) (PL 2417) cell culture containers to transduce stem cells with a key missing gene to create a treatment for X-linked severe combined immunodeficiency (SCID).
Sangamo is also developing sequence-specific ZFP-Nucleases (ZFNs) for therapeutic gene correction as a treatment and possible cure for a variety of monogenic diseases such as X-linked severe combined immunodeficiency (X-linked SCID) and sickle cell anemia (SCA).