Y Li, Z Glass, M Huang, ZY Chen, Q Xu - Biomaterials, 2020 - Elsevier
The recently developed CRISPR/Cas9 technology has revolutionized the genome engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics …
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of …
Gene editing-based therapeutic strategies grant the power to override cell machinery and alter faulty genes contributing to disease development like cancer. Nowadays, the principal …
K Huang, D Zapata, Y Tang, Y Teng, Y Li - Biomaterials, 2022 - Elsevier
Since its mechanism discovery in 2012 and the first application for mammalian genome editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …
P Germino-Watnick, M Hinds, A Le, R Chu, X Liu… - Cells, 2022 - mdpi.com
Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …
I Ates, T Rathbone, C Stuart, PH Bridges, RN Cottle - Genes, 2020 - mdpi.com
Impressive therapeutic advances have been possible through the advent of zinc-finger nucleases and transcription activator-like effector nucleases. However, discovery of the …
CRISPR/Cas9 system exploits the concerted action of Cas9 nuclease and programmable single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …
AB Soroka, SG Feoktistova, ON Mityaeva… - International Journal of …, 2023 - mdpi.com
In contrast to the standard enzyme-replacement therapy, administered from once per 7–14 days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …
L Wang, Y Yang, CA Breton, J White… - Blood, The Journal …, 2019 - ashpublications.org
Many genetic diseases, including hemophilia, require long-term therapeutic effects. Despite the initial success of liver-directed adeno-associated virus (AAV) gene therapy for …