Zobrazeno 1 - 10
of 332
pro vyhledávání: '"Roland W Herzog"'
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-14 (2024)
Abstract Current gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for striated muscles as it lacks many important functional domains o
Externí odkaz:
https://doaj.org/article/99a49e53f4014a01a4aca707caf156f6
Autor:
George Q Perrin, Irene Zolotukhin, Alexandra Sherman, Moanaro Biswas, Ype P de Jong, Cox Terhorst, Andrew M Davidoff, Roland W Herzog
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
The tolerogenic hepatic microenvironment impedes clearance of viral infections but is an advantage in viral vector gene transfer, which often results in immune tolerance induction to transgene products. Although the underlying tolerance mechanism has
Externí odkaz:
https://doaj.org/article/e8fcc8b2b0814e77b149f37b5fbe352e
Autor:
Phillip A Doerfler, Sushrusha Nayak, Manuela Corti, Laurence Morel, Roland W Herzog, Barry J Byrne
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA) deficiency). Unfortunately, the introduction of GAA and viral vectors encoding the enzyme can lead
Externí odkaz:
https://doaj.org/article/d33f7b4f31044bd883a85f17c966f1d6
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Therapeutic gene transfer holds the promise of providing lasting therapies and even cures for diseases that were previously untreatable or for which only temporary or suboptimal treatments were available. For some time, clinical gene therapy was char
Externí odkaz:
https://doaj.org/article/3869457b10a644a5963e0b8ed9e48b6e
Autor:
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively. Gene therapy for hemophilia with adeno-associated virus vectors has shown efficacy in hemophilia B
Externí odkaz:
https://doaj.org/article/cc6eb2caf37342bfa894aa969350dfa7
Ex vivo expanded autologous polyclonal regulatory T cells suppress inhibitor formation in hemophilia
Autor:
Debalina Sarkar, Moanaro Biswas, Gongxian Liao, Howard R Seay, George Q Perrin, David M Markusic, Brad E Hoffman, Todd M Brusko, Cox Terhorst, Roland W Herzog
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Adoptive cell therapy utilizing ex vivo expanded polyclonal CD4+CD25+FOXP3+ regulatory T cells (Treg) is in use in clinical trials for the treatment of type 1 diabetes and prevention of graft versus host disease in bone marrow transplantation. Here,
Externí odkaz:
https://doaj.org/article/6e375a589b1f4e66989e48a1fe42c693
Autor:
Sushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, Denise D Cloutier, Richie Khanna, Ken J Valenzano, Roland W Herzog, Barry J Byrne
Publikováno v:
PLoS ONE, Vol 9, Iss 6, p e98336 (2014)
Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA approved therapy for Pompe disease. Without ERT, severely affected individuals (early onset) succumb to the disease within 2 years of life. A spectrum
Externí odkaz:
https://doaj.org/article/c18505096a00432ab147b4446c7d4dfe
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Gene replacement therapies, like organ and cell transplantation, are likely to introduce neoantigens that elicit rejection via humoral and/or effector T-cell immune responses. Nonetheless, thanks to an ever-growing body of preclinical studies; it is
Externí odkaz:
https://doaj.org/article/3c589ab0a9ce47528d80876ecd846c6b
Autor:
Kasun Kodippili, Chady H. Hakim, Matthew J. Burke, Yongping Yue, James A. Teixeira, Keqing Zhang, Gang Yao, Gopal J. Babu, Roland W. Herzog, Dongsheng Duan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 2, Pp 101268- (2024)
Excessive cytosolic calcium accumulation contributes to muscle degeneration in Duchenne muscular dystrophy (DMD). Sarco/endoplasmic reticulum calcium ATPase (SERCA) is a sarcoplasmic reticulum (SR) calcium pump that actively transports calcium from t
Externí odkaz:
https://doaj.org/article/8999a19b16ac45ccb94ac3fd43620ab2
Publikováno v:
PLoS ONE, Vol 8, Iss 5, p e64342 (2013)
Viral vectors remain the most efficient and popular in deriving induced pluripotent stem cells (iPSCs). For translation, it is important to silence or remove the reprogramming factors after induction of pluripotency. In this study, we design an excis
Externí odkaz:
https://doaj.org/article/fa6a782ca870493b81d10b48967a0b15