Sentences with phrase «for severe combined immunodeficiency»

Cuvelier GD, Schultz KR, Davis J, Hirschfeld AF, Junker AK, Tan R, Turvey SE.: Optimizing outcomes of hematopoietic stem cell transplantation for severe combined immunodeficiency.

(In the early 2000s, five children who participated in a retrovirus - based gene therapy trial for severe combined immunodeficiency developed leukemia.)

Not exact matches

In California, state law requires that the blood be tested for about 80 diseases, including cystic fibrosis, severe combined immunodeficiency and primary congenital hypothyroidism.

Notably, research groups might be able to apply the approach described in this study to develop treatments for other blood diseases such as β - thalassemia, severe combined immunodeficiency (SCID), chronic granulomatous disease, rare disorders like Wiskott - Aldrich syndrome and Fanconi anemia, and even HIV infection.

A trial of a gene treatment for children with severe combined immunodeficiency, who have no functioning immune system, was called off after some of the patients developed leukaemia.

Fischer, in 2000, reported demonstrating the clinical efficacy of gene therapy for the first time, using blood stem cells to treat a fatal genetic disorder called X-linked severe combined immunodeficiency.

According to W. French Anderson, a gene therapy researcher at the University of Southern California, severe combined immunodeficiency (SCID) was a logical target for gene therapy.

Doctors at a hospital in Houston, Texas, managed to keep a boy born with «severe combined immunodeficiency» (SCID) alive for this long.

These cells are easily accessible (therefore, they are the first ones used in the clinic), seemingly well suited for gene transfer, and used in practice with new genes to correct two types of severe combined immunodeficiency (which are briefly described in the book).

This is in addition to the licensing of products such as GlaxoSmithKline's Strimvelis ex-vivo stem - cell therapy for treatment of severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA - SCID) in 2016 (1)-- has led to an increase in demand for therapeutic vector manufacturing capabilities.

St. Jude has played a key role in vector development, pioneering innovative vector designs for patients with hemophilia and the devastating immune disorder X-linked severe combined immunodeficiency (SCID).

Read about the St. Jude trial: LVXSCID - ND: Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants

LA JOLLA — For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal.

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.

The LVSCID clinical trial for X-linked severe combined immunodeficiency uses a lentiviral vector to insert a healthy copy of the IL2RG gene into blood cells.

For example, in the rare disease of Severe Combined Immunodeficiency a dam will be perfectly healthy but will pass on to fifty percent of her puppies the X gene with the recessive gene for the diseaFor example, in the rare disease of Severe Combined Immunodeficiency a dam will be perfectly healthy but will pass on to fifty percent of her puppies the X gene with the recessive gene for the diseafor the disease.

AUTOIMMUNE SYSTEM See GENETICS: Inherited Autoimmune Disorders for the following: Allergies / Atopy Autoimmune Hypothyroidism Bullous Pemphigold Compliment Deficiency Lupus Erythematosus Selective Iga Deficiency Severe Combined Immunodeficiency Thrombocytopenia.