Zobrazeno 1 - 10
of 173
pro vyhledávání: '"Charles A. Gersbach"'
Autor:
Mercedes Barzi, Tong Chen, Trevor J. Gonzalez, Francis P. Pankowicz, Seh Hoon Oh, Helen L. Streff, Alan Rosales, Yunhan Ma, Sabrina Collias, Sarah E. Woodfield, Anna Mae Diehl, Sanjeev A. Vasudevan, Thao N. Galvan, John Goss, Charles A. Gersbach, Beatrice Bissig-Choisat, Aravind Asokan, Karl-Dimiter Bissig
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-10 (2024)
Abstract Clinical translation of AAV-mediated gene therapy requires preclinical development across different experimental models, often confounded by variable transduction efficiency. Here, we describe a human liver chimeric transgene-free Il2rg −/
Externí odkaz:
https://doaj.org/article/3464bdb424c442108002dbd891da2d8d
Autor:
Jounghyun H. Lee, Kevin L. Shores, Jason J. Breithaupt, Caleb S. Lee, Daniella M. Fodera, Jennifer B. Kwon, Adarsh R. Ettyreddy, Kristin M. Myers, Benny J. Evison, Alexandra K. Suchowerska, Charles A. Gersbach, Kam W. Leong, George A. Truskey
Publikováno v:
APL Bioengineering, Vol 7, Iss 4, Pp 046103-046103-15 (2023)
Atherosclerosis is a primary precursor of cardiovascular disease (CVD), the leading cause of death worldwide. While proprotein convertase subtilisin/kexin 9 (PCSK9) contributes to CVD by degrading low-density lipoprotein receptors (LDLR) and altering
Externí odkaz:
https://doaj.org/article/f6d0b5cc6b244d04bac23d3123f01c78
Autor:
Trevor J. Gonzalez, Katherine E. Simon, Leo O. Blondel, Marco M. Fanous, Angela L. Roger, Maribel Santiago Maysonet, Garth W. Devlin, Timothy J. Smith, Daniel K. Oh, L. Patrick Havlik, Ruth M. Castellanos Rivera, Jorge A. Piedrahita, Mai K. ElMallah, Charles A. Gersbach, Aravind Asokan
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-17 (2022)
Abstract Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. Effective clinical translation is confounded, at least in part
Externí odkaz:
https://doaj.org/article/d9d15ee20e5e46a9b86982294d7e7093
Autor:
Chady H. Hakim, Sandeep R. P. Kumar, Dennis O. Pérez-López, Nalinda B. Wasala, Dong Zhang, Yongping Yue, James Teixeira, Xiufang Pan, Keqing Zhang, Emily D. Million, Christopher E. Nelson, Samantha Metzger, Jin Han, Jacqueline A. Louderman, Florian Schmidt, Feng Feng, Dirk Grimm, Bruce F. Smith, Gang Yao, N. Nora Yang, Charles A. Gersbach, Shi-jie Chen, Roland W. Herzog, Dongsheng Duan
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-12 (2021)
The Cas9-specific T cell response has been speculated to impair CRISPR therapy. Here, the authors show that local and systemic AAV CRISPR therapy induces cytotoxic killing and eliminates rescued dystrophin in canine models of Duchenne muscular dystro
Externí odkaz:
https://doaj.org/article/fa789cec57864e868cf64db8cae359c5
Branched-chain α-ketoacids are preferentially reaminated and activate protein synthesis in the heart
Autor:
Jacquelyn M. Walejko, Bridgette A. Christopher, Scott B. Crown, Guo-Fang Zhang, Adrian Pickar-Oliver, Takeshi Yoneshiro, Matthew W. Foster, Stephani Page, Stephan van Vliet, Olga Ilkayeva, Michael J. Muehlbauer, Matthew W. Carson, Joseph T. Brozinick, Craig D. Hammond, Ruth E. Gimeno, M. Arthur Moseley, Shingo Kajimura, Charles A. Gersbach, Christopher B. Newgard, Phillip J. White, Robert W. McGarrah
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-14 (2021)
Systemic modulation of branched-chain keto acid (BCKA) metabolism alters cardiac health. Here, the authors define the major fates of BCKA in the heart and demonstrate that acute exposure to BCKA levels found in obesity activates cardiac protein synth
Externí odkaz:
https://doaj.org/article/13e6e6480fe7436f81d6416509424af3
Autor:
Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 19, Iss , Pp 320-329 (2020)
Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies. Although certain AAV serotypes efficiently target muscle fibers, tra
Externí odkaz:
https://doaj.org/article/027bf4f182644f8e99d5b59e596aa1d1
Autor:
Karen Bulaklak, Charles A. Gersbach
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-4 (2020)
Gene therapy is at an inflection point. Recent successes in genetic medicine have paved the path for a broader second wave of therapies and laid the foundation for next-generation technologies. This comment summarizes recent advances and expectations
Externí odkaz:
https://doaj.org/article/e73be1499a3f43d1abd476b0fd01fb8c
Autor:
Amanda Dicks, Chia-Lung Wu, Nancy Steward, Shaunak S. Adkar, Charles A. Gersbach, Farshid Guilak
Publikováno v:
Stem Cell Research & Therapy, Vol 11, Iss 1, Pp 1-14 (2020)
Abstract Background Articular cartilage shows little or no capacity for intrinsic repair, generating a critical need of regenerative therapies for joint injuries and diseases such as osteoarthritis. Human-induced pluripotent stem cells (hiPSCs) offer
Externí odkaz:
https://doaj.org/article/f36c5cf155da46bd94d57c44b06c5220
Autor:
Brian C. Evans, R. Brock Fletcher, Kameron V. Kilchrist, Eric A. Dailing, Alvin J. Mukalel, Juan M. Colazo, Matthew Oliver, Joyce Cheung-Flynn, Colleen M. Brophy, John W. Tierney, Jeffrey S. Isenberg, Kurt D. Hankenson, Kedar Ghimire, Cynthia Lander, Charles A. Gersbach, Craig L. Duvall
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-19 (2019)
Most reagents designed to deliver cargo into cells are cationic and so cannot deliver cationic cargo. Here the authors show that pretreating cells with the anionic polymer poly(propylacrylic acid) facilitates the uptake and endosomal escape of a wide
Externí odkaz:
https://doaj.org/article/527a6ef95d124979b5fd95a610cb9fb1
Autor:
Joshua B. Black, Sean R. McCutcheon, Shataakshi Dube, Alejandro Barrera, Tyler S. Klann, Grayson A. Rice, Shaunak S. Adkar, Scott H. Soderling, Timothy E. Reddy, Charles A. Gersbach
Publikováno v:
Cell Reports, Vol 33, Iss 9, Pp 108460- (2020)
Summary: Technologies to reprogram cell-type specification have revolutionized the fields of regenerative medicine and disease modeling. Currently, the selection of fate-determining factors for cell reprogramming applications is typically a laborious
Externí odkaz:
https://doaj.org/article/e5a854fcace44b25a18afb8d37a05c63