Zobrazeno 1 - 10
of 283
pro vyhledávání: '"Timothy A. Nichols"'
Autor:
Bhavya S. Doshi, Benjamin J. Samelson-Jones, Timothy C. Nichols, Elizabeth P. Merricks, Joshua L. Siner, Robert A. French, Ben J. Lee, Valder R. Arruda, Mary Beth Callan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101205- (2024)
The hemophilias are the most common severe inherited bleeding disorders and are caused by deficiency of clotting factor (F) VIII (hemophilia A) or FIX (hemophilia B). The resultant bleeding predisposition significantly increases morbidity and mortali
Externí odkaz:
https://doaj.org/article/b7c59031eecd4b9ca3b2c7a6fcb6f7d7
Autor:
Lucas Van Gorder, Bhavya S. Doshi, Elinor Willis, Timothy C. Nichols, Emma Cook, John K. Everett, Elizabeth P. Merricks, Valder R. Arruda, Frederic D. Bushman, Mary Beth Callan, Benjamin J. Samelson-Jones
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101159- (2023)
Adeno-associated viral (AAV) vectors have traditionally been viewed as predominantly nonintegrating, with limited concerns for oncogenesis. However, accumulating preclinical data have shown that AAV vectors integrate more often than previously apprec
Externí odkaz:
https://doaj.org/article/c962f9d134214b5e82348ed40acae6dc
Autor:
Wen Yan, Chi‐Yeh Chung, Tao Xie, Mark Ozeck, Timothy C. Nichols, Jessica Frey, Akshata R. Udyavar, Shikhar Sharma, Thomas A. Paul
Publikováno v:
Molecular Oncology, Vol 16, Iss 6, Pp 1309-1328 (2022)
Small‐cell lung cancer (SCLC) is a heterogeneous disease, consisting of intratumoral and intertumoral neuroendocrine (ASCL1 and/or NEUROD1), mesenchymal‐like, and YAP‐driven transcriptional states. Lysine‐specific demethylase 1 (LSD1; also kn
Externí odkaz:
https://doaj.org/article/11d955d3d76647be8e32ac0b3418018d
Autor:
Junjiang Sun, Xiaojing Chen, Zheng Chai, Hongqian Niu, Amanda L. Dobbins, Timothy C. Nichols, Chengwen Li
Publikováno v:
Frontiers in Medicine, Vol 9 (2022)
Adeno-associated virus (AAV) gene therapy has been successfully applied in hemophilia patients excluding patients with inhibitors. During the coagulation pathway, activated factor V (FVa) functions downstream as a cofactor of activated factor X (FXa)
Externí odkaz:
https://doaj.org/article/f3625b459d5944d4ad50d94f43732a5c
Autor:
Xiaolei Pei, Wenwei Shao, Allene Xing, Charles Askew, Xiaojing Chen, Caibin Cui, Yasmina L. Abajas, David A. Gerber, Elizabeth P. Merricks, Timothy C. Nichols, Wuping Li, R. Jude Samulski, Chengwen Li
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 259-268 (2020)
Adeno-associated virus (AAV) vectors have been successfully used in patients with bleeding disorders and blindness. For human liver targeting, two major factors restrict effective AAV transduction after systemic administration of AAV vectors: human h
Externí odkaz:
https://doaj.org/article/ffd1eccee8e6456aa953cf4176aa8c2d
Autor:
Wenwei Shao, Junjiang Sun, Xiaojing Chen, Amanda Dobbins, Elizabeth P. Merricks, R. Jude Samulski, Timothy C. Nichols, Chengwen Li
Publikováno v:
Frontiers in Pharmacology, Vol 13 (2022)
Adeno-associated virus (AAV) mediated gene therapy has been successfully applied in clinical trials, including hemophilia. Novel AAV vectors have been developed with enhanced transduction and specific tissue tropism. Considering the difference in eff
Externí odkaz:
https://doaj.org/article/2afe3a173d2f462ca9c1fcf4aa273b71
Publikováno v:
PLoS ONE, Vol 15, Iss 10, p e0240896 (2020)
IntroductionThe rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B. Subcutaneous administration has historically been limited by low bioavailability and poten
Externí odkaz:
https://doaj.org/article/8c741e748a044c79abe6c6f6c0a7ee4e
Autor:
Junjiang Sun, Wenwei Shao, Xiaojing Chen, Elizabeth P. Merricks, Lauren Wimsey, Yasmina L. Abajas, Glenn P. Niemeyer, Clinton D. Lothrop, Paul E. Monahan, R. Jude Samulski, Timothy C. Nichols, Chengwen Li
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 257-267 (2018)
Adeno-associated virus (AAV) vectors have been successfully applied in hemophilia clinical trials. However, this approach is limited to patients without AAV-neutralizing antibodies (NAbs). In this study, we explored the feasibility of AAV re-administ
Externí odkaz:
https://doaj.org/article/ddd9feb7fb3a4072a978a554d53586c5
Autor:
Robert A. French, Benjamin J. Samelson-Jones, Glenn P. Niemeyer, Clinton D. Lothrop, Jr, Elizabeth P. Merricks, Timothy C. Nichols, Valder R. Arruda
Publikováno v:
Blood Advances, Vol 2, Iss 5, Pp 505-508 (2018)
Externí odkaz:
https://doaj.org/article/586745486b12485bae8ac688cf833cfb
Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Autor:
David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava, Roland W. Herzog
Publikováno v:
Journal of Translational Medicine, Vol 15, Iss 1, Pp 1-11 (2017)
Abstract Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have also identified several
Externí odkaz:
https://doaj.org/article/f621a02988f943ae9689ad35be3ac336