There, they began to specialize, creating all of the secondary or «daughter» cells that such stem cells normally produce — including healthy T cells with functioning
ADA genes.
Before Katlyn showed up at NIH, the doctors there were already well prepared: They had inserted healthy human
ADA genes into a modified mouse retrovirus — a type of virus that can enter human cells and transfer new genetic material right into the DNA strands in their nuclei.
Instead of using white blood cells, the British researchers, working with a Dutch group, want to take stem cells from bone marrow and insert
the ADA gene into these.
Stem cells were harvested from Nina's bone marrow and given a working version of
the ADA gene, before being injected back in.
Immune cells from 4 - year - old Ashanti DeSilva are given working versions of
the ADA gene to treat severe combined immunodeficiency disorder (SCID).
Researchers inserted
the ADA gene into the cells with the help of a retrovirus, which naturally inserts its genetic material into that of any cell it infects.
Not exact matches
So we saw the Greater New York Taxi Association, not generally known for its good works (
Gene Freidman's bio on the GNYTA website lists the «Israeli Defense Force» as one of his favorite charities), suing the City over
ADA requirements, over its failure to allow for hybrid vehicles as stipulated by the City Council, etc etc..
Her specific condition, called
ADA - SCID, had long tantalized researchers seeking to repair genetic defects with a technique called
gene therapy.
The first people to be treated with a
gene therapy had
ADA - SCID, also called «bubble boy disease», and some later got leukaemia, probably because the virus carrying the new
genes also switched on cancer
genes.
Jonathan Appleby, GSK project leader for the
ADA - SCID trial and head of cell and
gene therapy at the rare diseases unit, says there are many differences between working on this kind of trial and working with one for a common disease.
There are now more than 100
gene therapy clinical trials aimed toward cancer, genetic diseases (such as
ADA deficiency, cystic fibrosis and hemophilia A), infectious diseases (including AIDS) and autoimmune diseases (such as rheumatoid arthritis).
The unit is currently working on bringing to market a new
gene therapy for the immune disease
ADA - SCID, which is thought to affect only 350 children worldwide.
Richard Mulligan and his colleagues at the Massachusetts Institute of Technology and the Whitehead Institute for Biomedical Research in Cambridge, Massachusetts, introduced the
gene coding for
ADA into mouse cells.
Aiuti A, Roncarolo MG, Naldini L.
Gene therapy for ADA - SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal produ
Gene therapy for
ADA - SCID, the first marketing approval of an ex vivo
gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal produ
gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.
Clinical research activities are focused on the implementation of experimental protocols based on
gene or cell therapy for the treatment of genetic blood disorders such as
ADA - SCID, Wiskott - Aldrich Syndrome, Metachromatic Leukodystrophy, Beta - thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).
The successful results obtained with
ADA - SCID provided a rationale for extending the HSC
gene therapy approach to other diseases.
In subjects without the ApoE4
gene, compared to placebo, the MCT drink resulted in improved scores on the Alzheimer's Disease Assessment Scale - Cognitive Subscale (
ADAS - cog)(5).