Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Christopher R. Richmond"'
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 10, Iss , Pp 122-130 (2018)
Progress in gene editing research has been accelerated by utilizing engineered nucleases in combination with induced pluripotent stem cell (iPSC) technology. Here, we report transcription activator-like effector nuclease (TALEN)-mediated reincorporat
Externí odkaz:
https://doaj.org/article/5c25580fe484488fb2b78edcd83ed99d
Autor:
Karlaina J.L. Osmon, Patrick Thompson, Evan Woodley, Jagdeep S. Walia, Steven J. Gray, Zhilin Chen, Christopher R. Richmond
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 12, Iss, Pp 47-57 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
GM2 gangliosidoses are a family of severe neurodegenerative disorders resulting from a deficiency in the β-hexosaminidase A enzyme. These disorders include Tay-Sachs disease and Sandhoff disease, caused by mutations in the HEXA gene and HEXB gene, r
Publikováno v:
Molecular Therapy. Nucleic Acids
Molecular Therapy: Nucleic Acids, Vol 10, Iss, Pp 122-130 (2018)
Molecular Therapy: Nucleic Acids, Vol 10, Iss, Pp 122-130 (2018)
Progress in gene editing research has been accelerated by utilizing engineered nucleases in combination with induced pluripotent stem cell (iPSC) technology. Here, we report transcription activator-like effector nuclease (TALEN)-mediated reincorporat
Autor:
Colin D. Funk, A. Elizabeth de Guzman, Laurel L. Ballantyne, Brian J. Nieman, Christopher R. Richmond, Nader Ghasemlou
Publikováno v:
Neurochemistry International. 145:104984
Arginase-1 (Arg1) is an enzyme controlling the final step of the urea cycle, with highest expression in the liver and lower expression in the lungs, pancreas, kidney, and some blood cells. Arg1 deficiency is an inherited urea cycle disorder presentin