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searine 2 days ago

The tech behind this is not new or difficult. The issues are related to safety and regulation. Early efforts in gene therapy had disastrous results and current treatments are not trying to repeat past mistakes.

There is tremendous potential for gene therapy to cure disease, however it needs (and so far has had) strict regulation, particularly if the changes can be inherited.

beambot 2 days ago | parent [-]

> Early efforts in gene therapy had disastrous results

Can you share examples...? Just curious as an outsider looking in.

ortusdux 2 days ago | parent [-]

https://pharmaceutical-journal.com/article/feature/gene-ther...

"The most notable obstacle faced in the gene therapy field was that of Gelsinger in 1999, who is understood to have died after his body overreacted to the adenovirus vector. Gelsinger had a rare disorder in which the liver lacks a functional copy of the ornithine transcarbamylase (OTC) gene and, consequently, the body is unable to eliminate ammonia, a toxic breakdown product of protein metabolism."

"A gene therapy for children with severe combined immunodeficiency (SCID) was delivered [to two independent groups in London and Paris] and was incredibly successful,” explains Griesenbach. “But, [in 2008; between three and six years later], a small proportion of children [in Paris] developed leukaemia induced by the vector, which had inserted itself into a gene that controls cell division [4] .”