Genetics

A hiccup in gene therapy progress?

Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...

Genetics

AAV gene delivery vectors and cancer—The debate continues

Overwhelming evidence from the biomedical literature shows that adeno-associated virus 2 (AAV2), a viral vector often used to deliver therapeutic genes, is not associated with cancer and, in fact, may protect against cancer. ...

Genetics

Early gene therapy results in Wiskott-Aldrich syndrome promising

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott-Aldrich syndrome (WAS), a life-threatening genetic blood and immune disorder, at the 57th annual ...

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