Human Genetics: Critically discuss the applications of gene therapy in the treatment of SMA and the results obtained in clinical trials, including the advantages and disadvantages of in vivo gene therapy administration
Human Genetics Spinal Muscular Atrophy (SMA) is a rare and invalidating monogenic disease with an incidence of 1 in 10,000 births and a low life expectancy. In March 2021, the NHS approved the use of an in vivo gene replacement therapy treatment for babies diagnosed with SMA up to two years of age, making it … Read more