Question

The first reported case of gene therapy was in 1999 5. O b) by Dr. Jennifer Doudna using CRISPR-Cas9 O a) and was done to Jesse Gelsinger, an 18- on herself year-old with an X-linked genetic disease involving the metabolism of ammonia. O c) by Chinese scientist Jianku He to embryos to O d) by Dr. Victoria Ashton on a patient with cystic disable their HIV receptors fibrosis

Answer 1

The answer to the question is (a) .

The first reported case of gene therapy was in 1999 and was done to Jesse Gelsinger, an 18 year old (1981-1999) who was suffering from ornithine transcarbamylase deficiency which is a X-linked genetic disease of the liver. Its symptoms include an inability to metabolize ammonia.

He had joined the clinical trial run by University of Pennsylvania where he was injected with adenoviral vector containing the corrected gene. However, he died four days later owing to a massive immune response triggered by injection of viral vector which led to multiple organ failure and ultimately brain death.

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