AP BIOLOGY

Detailed explanation-1: -Gene Therapy was initially meant to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy (see video 2) and sickle cell anemia (see also Wiley database on indications addressed by gene therapy clinical trials).

Detailed explanation-2: -in England, people with CF were given a dose of a non-integrating gene therapy once per month for a year. The study indicated that the CF gene therapy was safe and resulted in a small improvement in lung function.

Detailed explanation-3: -Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways.

Detailed explanation-4: -Gene therapy has already shown amazing potential in the treatment and management of PD. The current medication and/or surgical options available have varying efficacy with time, and with no cure available action needs to be taken.

Detailed explanation-5: -Gene therapy for Alzheimer’s disease is unique in that it can essentially circumvent the BBB by delivering genes directly via delivery systems, like viral vectors. Additionally, due to the persistent nature of gene expression, only a single administration of the therapy is theoretically required.