Progress in and Prospects of Genome Editing Tools for Human Disease Model Development and Therapeutic Applications
Programmable nucleases, such as zinc finger nucleases (ZFNs), transcription activator-like
effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats …
effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats …
Rare genetic disorders in India: Current status, challenges, and CRISPR-based therapy
P Bhattacharyya, K Mehndiratta, S Maiti… - Journal of …, 2024 - Springer
Rare genetic diseases are a group of life-threatening disorders affecting significant
populations worldwide and posing substantial challenges to healthcare systems globally …
populations worldwide and posing substantial challenges to healthcare systems globally …
Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
S Muñoz, J Bertolin, V Jimenez, ML Jaén, M Garcia… - Molecular …, 2024 - Elsevier
Objective Pompe disease (PD) is caused by deficiency of the lysosomal enzyme acid α-
glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy …
glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy …
Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy
ML Zhang, HB Li, Y Jin - Frontiers in Genetics, 2024 - frontiersin.org
The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9
nuclease system has been extensively used for genome editing and gene modification in …
nuclease system has been extensively used for genome editing and gene modification in …