Zobrazeno 1 - 10
of 92
pro vyhledávání: '"John T Gray"'
Autor:
Stephanie A Shumar, Paolo Fagone, Adolfo Alfonso-Pecchio, John T Gray, Jerold E Rehg, Suzanne Jackowski, Roberta Leonardi
Publikováno v:
PLoS ONE, Vol 10, Iss 6, p e0130013 (2015)
Pantothenate kinase-associated neurodegeneration, PKAN, is an inherited disorder characterized by progressive impairment in motor coordination and caused by mutations in PANK2, a human gene that encodes one of four pantothenate kinase (PanK) isoforms
Externí odkaz:
https://doaj.org/article/015a4f62aa274b6580709bbc9c4775a8
Publikováno v:
PLoS ONE, Vol 8, Iss 4, p e62333 (2013)
Hematopoietic stem cell gene therapy requires the use of integrating retroviral vectors in order to stably transmit a therapeutic gene to mature blood cells. Human clinical trials have shown that some vector integration events lead to disrupted regul
Externí odkaz:
https://doaj.org/article/9ea4f24a3353421699f3e94dd0ed3a03
Autor:
Mark A. Brimble, Pei-Hsin Cheng, Stephen M. Winston, Isaiah L. Reeves, Aisha Souquette, Yunyu Spence, Junfang Zhou, Yong-Dong Wang, Christopher L. Morton, Marcus Valentine, Paul G. Thomas, Amit C. Nathwani, John T. Gray, Andrew M. Davidoff
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 280-291 (2022)
Recombinant adeno-associated virus (rAAV) vectors are increasingly being used for clinical gene transfer and have shown great potential for the treatment of several monogenic disorders. However, contaminant DNA from producer plasmids can be packaged
Externí odkaz:
https://doaj.org/article/9ce90bd19a374a6086d2a19259d8a399
Autor:
Jenny A. Greig, Jayme M.L. Nordin, Christine Draper, Deirdre McMenamin, Edward A. Chroscinski, Peter Bell, John T. Gray, Laura K. Richman, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 237-244 (2018)
Liver metabolism disorders are attractive targets for gene therapy, because low vector doses can reverse the buildup of toxic metabolites in the blood. Crigler-Najjar syndrome is an inherited disorder of bilirubin metabolism that is caused by the abs
Externí odkaz:
https://doaj.org/article/0cdfa4f8c1e84e47b68f369400bd39cb
Autor:
Laura K. Richman, Jayme M.L. Nordin, Peter Bell, Edward A. Chroscinski, James M. Wilson, Jenny A. Greig, Christine Draper, John T. Gray, Deirdre McMenamin
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss, Pp 237-244 (2018)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Liver metabolism disorders are attractive targets for gene therapy, because low vector doses can reverse the buildup of toxic metabolites in the blood. Crigler-Najjar syndrome is an inherited disorder of bilirubin metabolism that is caused by the abs
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b5a2b5980f4afed874d4de0c4b406610
https://doi.org/10.1016/j.omtm.2018.07.008
https://doi.org/10.1016/j.omtm.2018.07.008
Autor:
Suyash Prasad, Tim Stinchcombe, Salvador Rico, Christina Chaivorapol, Emma James, Jacqueline Brassard, John T. Gray, Garrett Heffner, Fulvio Mavilio, Justine Cunningham, Michelle Eggers
Publikováno v:
Molecular Genetics and Metabolism. 129:S106
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::b80f945bb50a0a54ce78fffd98782002
https://doi.org/10.1016/j.ymgme.2019.11.272
https://doi.org/10.1016/j.ymgme.2019.11.272
Autor:
David L. Mack, Jean-Baptiste Dupont, Ana Buj-Bello, Michael W. Lawlor, Robert W. Grange, John T. Gray, Jianjun Guo, Martin K. Childers
Multiple clinical trials employing recombinant adeno-associated viral (rAAV) vectors have been initiated for neuromuscular disorders, including Duchenne and limb-girdle muscular dystrophies, spinal muscular atrophy, and recently X-linked myotubular m
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4005986ac78bc21c9d069085cd04e9ae
https://doi.org/10.1101/499384
https://doi.org/10.1101/499384
Autor:
Vincent J Carson, Michael D Fox, Kevin A. Strauss, Michael W. Lawlor, Han-Zhong Feng, John T. Gray, Karlla W. Brigatti, J P Jin
We describe the natural history of ‘Amish’ nemaline myopathy (ANM), an infantile-onset, lethal disease linked to a pathogenic c.505G>T nonsense mutation of TNNT1, which encodes the slow fiber isoform of troponin T (TNNT1; a.k.a. TnT). The TNNT1 c
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d291c14cdf88ffb2ef1086937e2e0794
https://europepmc.org/articles/PMC6121192/
https://europepmc.org/articles/PMC6121192/
Autor:
Yimei Li, John T. Gray, C H Pui, Paul Eldridge, Duangchan Suwannasaen, Wing Keung Chan, Wing Leung, Robert E. Throm, Jim Houston
Publikováno v:
Leukemia
Chimeric antigen receptor (CAR)-redirected cellular therapy is an attractive modality for cancer treatment. We hypothesized that allogeneic CAR-engineered CD45RA-negative T cells can control cancer and infection without the risk of graft-versus-host
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2b925f279f4912d763dc3d8f06923cea
http://europepmc.org/articles/PMC4275423
http://europepmc.org/articles/PMC4275423
Publikováno v:
Molecular Genetics and Metabolism. 123:S55-S56
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::92d8312dbaf1c55d88492937d5d6fb00
https://doi.org/10.1016/j.ymgme.2017.12.132
https://doi.org/10.1016/j.ymgme.2017.12.132