Current landscape of gene-editing technology in biomedicine: Applications, advantages, challenges, and perspectives.

ABSTRACT: The expanding genome editing toolbox has revolutionized life science research ranging from the bench to the bedside. These "molecular scissors" have offered us unprecedented abilities to manipulate nucleic acid sequences precisely in living cells from diverse species. Continued advances in genome editing exponentially broaden our knowledge of human genetics, epigenetics, molecular biology, and pathology. Currently, gene editing-mediated therapies have led to impressive responses in patients with hematological diseases, including sickle cell disease and thalassemia. With the discovery of more efficient, precise and sophisticated gene-editing tools, more therapeutic gene-editing approaches will enter the clinic to treat various diseases, such as acquired immunodeficiency sydrome (AIDS), hema

SUBMITTER: Zhou W 

PROVIDER: S-EPMC9283854 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature