CRISPR RNA-Guided Gene Editing and its Clinical Research Applications in Hematology with Focus on Inherited Germline Predisposition to Hematologic Malignancies DOI Open Access
Rina Kansal

Опубликована: Июнь 10, 2024

Clustered regularly interspaced short palindromic repeats (CRISPR) based gene-editing has begun to transform the treatment landscape of genetic diseases. The history discovery CRISPR/CRISPR-associated (Cas) proteins/single guide RNA (sgRNA)-based since first report repetitive sequences unknown significance in 1987 is fascinating, instructive, and inspiring for future advances. recent approval CRISPR-Cas9-based gene therapy treat patients with severe sickle cell anemia transfusion-dependent beta thalassemia renewed hope treating other hematologic diseases, including germline predisposition malignancies, who would benefit greatly from development CRISPR-based therapies. purpose this manuscript three-fold: first, a chronological description CRISPR-Cas9-sgRNA-based editing; second, brief current state clinical research selected applications diseases therapy; third, progress therapies inherited bone marrow failure syndromes, hopefully stimulate efforts towards developing these syndromes conditions malignancies.

Язык: Английский

CRISPR-Based Genetic Improvement of Aquaculture Species DOI Creative Commons
Abdulkadir Bayır,

Selma Saoula,

Ammar Almansour

и другие.

Fishes, Год журнала: 2025, Номер 10(2), С. 84 - 84

Опубликована: Фев. 18, 2025

The clustered regularly interspaced short palindromic repeat-associated protein (CRISPR-Cas) system is considered a potential game-changer in the aquaculture sector. CRISPR-Cas9 derived from an adaptive mechanism of immune some prokaryotes. CRISPR/Cas9 potentially accelerates rate sustained genetic gain for and seafood production sectors. Unlike conventional genome-editing techniques, more cost-effective, user-friendly, extremely precise. It enables overcoming large-scale challenges aquaculture. Traits such as high fertility, external fertilization, shorter generation time, well-established breeding methods, ability to raise larvae offer benefits applying genome editing most aquacultural species. use precise where desired modifications are made target gene. There likelihood that intended alterations will be achieved, resulting transmission trait next generation. In this paper, we review how CRISPR evolved, its basic categories different Cas systems, well molecular CRISPR/Cas. We also highlight discuss applications CRISPR/Cas industry. Moreover, using technology briefly discussed. This pathway charts course future which has enable fulfill world food requirements with public ecological safety.

Язык: Английский

Процитировано

0
CRISPR RNA-Guided Gene Editing and its Clinical Research Applications in Hematology with Focus on Inherited Germline Predisposition to Hematologic Malignancies DOI Open Access
Rina Kansal

Опубликована: Июнь 10, 2024

Clustered regularly interspaced short palindromic repeats (CRISPR) based gene-editing has begun to transform the treatment landscape of genetic diseases. The history discovery CRISPR/CRISPR-associated (Cas) proteins/single guide RNA (sgRNA)-based since first report repetitive sequences unknown significance in 1987 is fascinating, instructive, and inspiring for future advances. recent approval CRISPR-Cas9-based gene therapy treat patients with severe sickle cell anemia transfusion-dependent beta thalassemia renewed hope treating other hematologic diseases, including germline predisposition malignancies, who would benefit greatly from development CRISPR-based therapies. purpose this manuscript three-fold: first, a chronological description CRISPR-Cas9-sgRNA-based editing; second, brief current state clinical research selected applications diseases therapy; third, progress therapies inherited bone marrow failure syndromes, hopefully stimulate efforts towards developing these syndromes conditions malignancies.

Язык: Английский

Процитировано

2