Tag Archives: Gene therapy

ITALY – Stem Cells. New Method to Combat Immunodeficiencies in Children at San Raffaele in Milan

Maria Grazia Roncarolo

It was stunning to see them closed inside of those plastic bubbles, kept far from all external contact because their immune system does not react against any foreign antigens. Today scientists can say that ADA-SCID (adenosine deaminase deficiency), a serious combined immunodeficiency caused due to a lack of the adenosine deaminase enzyme, has been definitively defeated by gene therapy developed at San Raffaele of Milan.

The final study, which combined the conclusions of clinical studies, which began in 2000 on strategic therapies developed by the HSR-TIGET (San Raffaele Telethon Institute for Gene Therapy) group, led
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Stem cell-gene therapy technique cures genetic disease in human cell line

Image by JohnnyRokkit via Flickr

Scientists are one step closer to creating a gene therapy/stem cell combination to combat genetic diseases. With work, this research may lead to not only curing the disease, but also repairing the damage left behind.

While gene therapy is a burgeoning field that has shown great results in treating genetic disorders, many of those diseases leave behind heavily damaged tissue that the body is unable to repair. So even if the disease is completely eradicated, quality of life may not necessarily improve, and without help, health can still continue to deteriorate.

Since stem cell research began, there
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9 babies healed by Italian gene therapy

Image via Wikipedia

It has been about 8 years since Salsabil, the first Palestinia baby suffering congenital immunodeficiency (Ada-Scid), has been healed using gene therapy. And it has been about 7 years since her story to Abdul Rahim’s one, a Pakistani baby born in the end of 2006 in Qatar.
His parents did lose 3 children because of Ada-Scid, and Abdul’s case is one of the most symbolic.
Just after his birth doctors did diagnose his illness and succeeded to healt his pneumonia during his first days of life.
So Abdul’s parents and doctors who were follow him contacted the San Raffaele Telethon
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Stanford stem cell experts underline flaws in drug making system

Academic institutions are in a much better position than pharmaceutical companies to make the best decisions about which therapies deserve further development. That was the underlying message from a pair of Stanford researchers at a panel on stem cell science at last weekend’s Association of Health Care Journalism 2015 conference.

“There’s an inherent flaw in our system,” said Irving Weissman, MD, director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine. “Companies are driven by the desire for profits rather than the desire to find the best therapy, and they often give up on discoveries too early.”

Weissman cited
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ITALY – Stem Cells. New Method to Combat Immunodeficiencies in Children at San Raffaele in Milan.

Image by pianetatschai via Flickr

It was stunning to see them closed inside of those plastic bubbles, kept far from all external contact because their immune system does not react against any foreign antigens. Today scientists can say that ADA-SCID (adenosine deaminase deficiency), a serious combined immunodeficiency caused due to a lack of the adenosine deaminase enzyme, has been definitively defeated by gene therapy developed at San Raffaele of Milan.

The final study, which combined the conclusions of clinical studies, which began in 2000 on strategic therapies developed by the HSR-TIGET (San Raffaele Telethon Institute for Gene Therapy) group,
Read More…