Abstract
Five distinct gene therapy approaches have been developed for treating AATD. These approaches include knockout of the mutant (PiZ) allele by introduction of double-strand breaks (DSBs) and subsequent creation of insertions and deletions (indels) by DSB repair, homology-directed repair (HDR) targeted to the mutation site, base editing, prime editing, and alternatively targeted knock-in techniques. Each approach will be discussed and a brief summary of a standard CRISPR–Cas9 targeting method will be presented.
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Gruntman, A.M., Xue, W., Flotte, T.R. (2024). Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency. In: Bristow, C.L. (eds) Alpha-1 Antitrypsin. Methods in Molecular Biology, vol 2750. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-3605-3_2
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DOI: https://doi.org/10.1007/978-1-0716-3605-3_2
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