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Nature Methods|July 15, 2020
Novel NanoLuc substrates enable bright two-population bioluminescence imaging in animalsYichi Su, Joel R Walker, Yunhee Park, et al.
Research and Practice in Thrombosis and Haemostasis|April 16, 2025
Development of a novel gene editing lexicon for hemophilia: methodology and resultsCraig M Kessler, Leonard A Valentino, Courtney D Thornburg, et al.
Circulation|July 19, 2019
Allele-Specific Silencing Ameliorates Restrictive Cardiomyopathy Attributable to a Human Myosin Regulatory Light Chain MutationKathia Zaleta-Rivera, Alexandra Dainis, Alexandre J S Ribeiro, et al.
Nature Communications|November 25, 2021
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine modelsChady H Hakim, Sandeep R P Kumar, Dennis O Pérez-López, et al.
Viruses|August 14, 2020
SARS-CoV-2 RNA Extraction Using Magnetic Beads for Rapid Large-Scale Testing by RT-qPCR and RT-LAMPSteffen Klein, Thorsten G Müller, Dina Khalid, et al.
American Journal of Human Genetics|August 5, 2020
Evolution of a Human-Specific Tandem Repeat Associated with ALSMeredith M Course, Kathryn Gudsnuk, Samuel N Smukowski, et al.
Science Advances|September 21, 2022
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disordersJihad El Andari, Edith Renaud-Gabardos, Warut Tulalamba, et al.
Blood|January 8, 2003
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia BCatherine S Manno, Amy J Chew, Sylvia Hutchison, et al.
Science Translational Medicine|November 28, 2014
Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosaVittorio Sebastiano, Hanson Hui Zhen, Bahareh Haddad, et al.
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