Therapeutic gene editing strategies using CRISPR-Cas9 for the β-hemoglobinopathies.

ABSTRACT: With advancements in gene editing technologies, our ability to make precise and efficient modifications to the genome is increasing at a remarkable rate, paving the way for scientists and clinicians to uniquely treat a multitude of previously irremediable diseases. CRISPR-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, is a gene editing platform with the ability to alter the nucleotide sequence of the genome in living cells. This technology is increasing the number and pace at which new gene editing treatments for genetic disorders are moving toward the clinic. The β-hemoglobinopathies are a group of monogenic diseases, which despite their high prevalence and chronic debilitating nature, continue to have few therapeutic options ava

SUBMITTER: Papizan JB 

PROVIDER: S-EPMC8038529 | biostudies-literature | 2020 Nov

REPOSITORIES: biostudies-literature