Zobrazeno 1 - 10
of 196
pro vyhledávání: '"Mark A. Behlke"'
Autor:
Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P. Dever, Omaira Alberquilla, Rebeca Sanchez-Dominguez, Joab Camarena, Isabel Ojeda-Perez, Mercedes Dessy-Rodriguez, Rolf Turk, Mollie S. Schubert, Annalisa Lattanzi, Liwen Xu, Jose L. Lopez-Lorenzo, Paola Bianchi, Juan A. Bueren, Mark A. Behlke, Matthew Porteus, Jose-Carlos Segovia
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 237-248 (2021)
Pyruvate kinase deficiency (PKD), an autosomal-recessive disorder, is the main cause of chronic non-spherocytic hemolytic anemia. PKD is caused by mutations in the pyruvate kinase, liver and red blood cell (PKLR) gene, which encodes for the erythroid
Externí odkaz:
https://doaj.org/article/36126901876d48f9918fa4a741965bd8
Autor:
Gavin Kurgan, Rolf Turk, Heng Li, Nathan Roberts, Garrett R. Rettig, Ashley M. Jacobi, Lauren Tso, Morgan Sturgeon, Massimo Mertens, Roel Noten, Kurt Florus, Mark A. Behlke, Yu Wang, Matthew S. McNeill
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 478-491 (2021)
CRISPR systems enable targeted genome editing in a wide variety of organisms by introducing single- or double-strand DNA breaks, which are repaired using endogenous molecular pathways. Characterization of on- and off-target editing events from CRISPR
Externí odkaz:
https://doaj.org/article/5e209aed7e8a4ef2be494827ee71b257
Autor:
Liyang Zhang, John A. Zuris, Ramya Viswanathan, Jasmine N. Edelstein, Rolf Turk, Bernice Thommandru, H. Tomas Rube, Steve E. Glenn, Michael A. Collingwood, Nicole M. Bode, Sarah F. Beaudoin, Swarali Lele, Sean N. Scott, Kevin M. Wasko, Steven Sexton, Christopher M. Borges, Mollie S. Schubert, Gavin L. Kurgan, Matthew S. McNeill, Cecilia A. Fernandez, Vic E. Myer, Richard A. Morgan, Mark A. Behlke, Christopher A. Vakulskas
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-15 (2021)
The utility of AsCas12a can be limited to poor editing efficiency. Here the authors identify a variant, “AsCas12a Ultra”, that has high on-target specificity demonstrated through editing of clinically relevant T cell genes.
Externí odkaz:
https://doaj.org/article/0168163683a24415a8a5f32e5a19e09a
Autor:
Jenny Shapiro, Ortal Iancu, Ashley M. Jacobi, Matthew S. McNeill, Rolf Turk, Garrett R. Rettig, Ido Amit, Adi Tovin-Recht, Zohar Yakhini, Mark A. Behlke, Ayal Hendel
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 1097-1107 (2020)
Genome editing of human cluster of differentiation 34+ (CD34+) hematopoietic stem and progenitor cells (HSPCs) holds great therapeutic potential. This study aimed to optimize on-target, ex vivo genome editing using the CRISPR-Cas9 system in CD34+ HSP
Externí odkaz:
https://doaj.org/article/74189cca19364566b5cb4597df8b7115
Autor:
Yoshitsugu Aoki, Cristina S. J. Rocha, Taavi Lehto, Shouta Miyatake, Henrik Johansson, Yasumasa Hashimoto, Joel Z. Nordin, Imre Mager, Misako Aoki, McClorey Graham, Chaitra Sathyaprakash, Thomas C. Roberts, Matthew J. A. Wood, Mark A. Behlke, Samir El Andaloussi
Publikováno v:
Frontiers in Physiology, Vol 12 (2021)
Splice-switching antisense oligonucleotide- (SSO-) mediated correction of framedisrupting mutation-containing premessenger RNA (mRNA) transcripts using exon skipping is a highly promising treatment method for muscular diseases such as Duchenne muscul
Externí odkaz:
https://doaj.org/article/0ea4440e5f7542ee82645e95690437a3
Autor:
John D. Lueck, Jae Seok Yoon, Alfredo Perales-Puchalt, Adam L. Mackey, Daniel T. Infield, Mark A. Behlke, Marshall R. Pope, David B. Weiner, William R. Skach, Paul B. McCray, Christopher A. Ahern
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-11 (2019)
Premature termination codon suppression therapy could be used to treat a range of genetic disorders. Here the authors present a high-throughput cell-based assay to identify anticodon engineered tRNAs with high suppression activity.
Externí odkaz:
https://doaj.org/article/407180d474f9434ca10313d5ef3ae933
Autor:
M. Kyle Cromer, Kiran R. Majeti, Garrett R. Rettig, Karthik Murugan, Gavin L. Kurgan, Nicole M. Bode, Jessica P. Hampton, Christopher A. Vakulskas, Mark A. Behlke, Matthew H. Porteus
Publikováno v:
Molecular Therapy. 31:1074-1087
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::82f779700be01951f9a084396b896b8a
https://doi.org/10.1016/j.ymthe.2023.02.011
https://doi.org/10.1016/j.ymthe.2023.02.011
Autor:
Rebecca L. Kow, Timothy J. Strovas, Pamela J. McMillan, Ashley M. Jacobi, Mark A. Behlke, Aleen D. Saxton, Caitlin S. Latimer, C. Dirk Keene, Brian C. Kraemer
Publikováno v:
Neurobiology of Disease, Vol 147, Iss , Pp 105148- (2021)
Aging drives pathological accumulation of proteins such as tau, causing neurodegenerative dementia disorders like Alzheimer's disease. Previously we showed loss of function mutations in the gene encoding the poly(A) RNA binding protein SUT-2/MSUT2 su
Externí odkaz:
https://doaj.org/article/a306260c5f1f44a8881453e339558866
Autor:
Peter Järver, Aleksandra Dondalska, Candice Poux, AnnSofi Sandberg, Joseph Bergenstråhle, Annette E. Sköld, Nathalie Dereuddre-Bosquet, Fréderic Martinon, Sandra Pålsson, Eman Zaghloul, David Brodin, Birgitta Sander, Kim A. Lennox, Mark A. Behlke, Samir EL-Andaloussi, Janne Lehtiö, Joakim Lundeberg, Roger LeGrand, Anna-Lena Spetz
Publikováno v:
Scientific Reports, Vol 8, Iss 1, Pp 1-17 (2018)
Abstract Recognition of nucleic acids by endosomal Toll-like receptors (TLR) is essential to combat pathogens, but requires strict control to limit inflammatory responses. The mechanisms governing this tight regulation are unclear. We found that sing
Externí odkaz:
https://doaj.org/article/8ee60f6cd9e941b7b20a1654b77636dc
Autor:
Rolen M. Quadros, Hiromi Miura, Donald W. Harms, Hisako Akatsuka, Takehito Sato, Tomomi Aida, Ronald Redder, Guy P. Richardson, Yutaka Inagaki, Daisuke Sakai, Shannon M. Buckley, Parthasarathy Seshacharyulu, Surinder K. Batra, Mark A. Behlke, Sarah A. Zeiner, Ashley M. Jacobi, Yayoi Izu, Wallace B. Thoreson, Lisa D. Urness, Suzanne L. Mansour, Masato Ohtsuka, Channabasavaiah B. Gurumurthy
Publikováno v:
Genome Biology, Vol 18, Iss 1, Pp 1-15 (2017)
Abstract Background Conditional knockout mice and transgenic mice expressing recombinases, reporters, and inducible transcriptional activators are key for many genetic studies and comprise over 90% of mouse models created. Conditional knockout mice a
Externí odkaz:
https://doaj.org/article/cc09dd43b09b4403b9ad7415943adf1e