Zobrazeno 1 - 10
of 91
pro vyhledávání: '"Thomas J. Conlon"'
Autor:
Thomas J. Conlon, Fulvio Mavilio
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 8, Iss C, Pp 181-182 (2018)
Externí odkaz:
https://doaj.org/article/0ed6f01a35774d5cb67a9596355604c7
Autor:
Sharla M. Birch, Michael W. Lawlor, Thomas J. Conlon, Lee-Jae Guo, Julie M. Crudele, Eleanor C. Hawkins, Peter P. Nghiem, Mihye Ahn, Hui Meng, Margaret J. Beatka, Brittany A. Fickau, Juan C. Prieto, Martin A. Styner, Michael J. Struharik, Courtney Shanks, Kristy J. Brown, Diane Golebiowski, Amanda K. Bettis, Cynthia J. Balog-Alvarez, Nathalie Clement, Kirsten E. Coleman, Manuela Corti, Xiufang Pan, Stephen D. Hauschka, J. Patrick Gonzalez, Carl A. Morris, Joel S. Schneider, Dongsheng Duan, Jeffrey S. Chamberlain, Barry J. Byrne, Joe. N. Kornegay
Publikováno v:
Science translational medicine. 15(677)
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene. Although mouse models of DMD provide insight into the potential of a corrective the
Autor:
Savitri Mandapati, Chunjuan Song, Alok K. Sharma, Mailin Van Hoosear, William W. Hauswirth, Guo-jie Ye, Jeffrey D. Chulay, Adrian M. Timmers, Cayrn Plummer, Ping Zhu, David R. Knop, Kari B. Green, Paulette M. Robinson, Peter Sonnentag, Wen-Tao Deng, Mark S. Shearman, Thomas J. Conlon, Kirsten E. Coleman
Publikováno v:
Human Gene Therapy Clinical Development. 29:188-197
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa
Autor:
Francisco Lourenco, Christina A. Pacak, Silveli Suzuki-Hatano, Chelsea Larkin, Kathryn Taggart, Amara H. Estrada, Luiz Bolfer, Thomas J. Conlon
Publikováno v:
Scientific Reports
Scientific Reports, Vol 10, Iss 1, Pp 1-11 (2020)
Scientific Reports, Vol 10, Iss 1, Pp 1-11 (2020)
A splice site mutation in the canine pyruvate dehydrogenase kinase 4 (PDK4) gene has been shown to be associated with the development of dilated cardiomyopathy (DCM) in Doberman Pinchers (DPs). Subsequent studies have successfully demonstrated the us
Autor:
Monika Dambska, Laurie M. Brown, Young Mok Lee, Kirsten E. Coleman, Andrew Specht, Kathryn R Dahlberg, David A. Weinstein, Thomas J. Conlon, Ana M. Estrella
Publikováno v:
Journal of Inherited Metabolic Disease. 41:977-984
Viral mediated gene therapy has progressed after overcoming early failures, and gene therapy has now been approved for several conditions in Europe and the USA. Glycogen storage disease (GSD) type Ia, caused by a deficiency of glucose-6-phosphatase-
Autor:
Thomas J. Conlon, Janet M. Benson, JeanClare Seagrave, John F. Engelhardt, William B. Guggino, Liudmila Cebotaru, Ziying Yan, Guangping Gao
Publikováno v:
Human Gene Therapy Clinical Development. 28:145-156
Cystic fibrosis (CF) is an autosomal recessive disease that is potentially treatable by gene therapy. Since the identification of the gene encoding CF transmembrane conductance regulator, a number of preclinical and clinical trials have been conducte
Autor:
Ingeborg M. Langohr, Dodd G. Sledge, Joshua T. Bartoe, András M. Komáromy, Thomas J. Conlon, Simon M. Petersen-Jones, Ryan F. Boyd, Sanford L. Boye, William W. Hauswirth, Shannon E. Boye, Kirsten E. Erger
Publikováno v:
Gene Therapy. 23:548-556
Adeno-associated virus (AAV) vector-based gene therapy is a promising treatment strategy for delivery of neurotrophic transgenes to retinal ganglion cells (RGCs) in glaucoma patients. Retinal distribution of transgene expression following intravitrea
Autor:
Rui Hou, Kirsten E. Erger, David G. Birch, Wenqiu Wang, Fowzan S. Alkuraya, William W. Hauswirth, Matthew M. LaVail, Douglas Vollrath, Fahad Al Saikhan, Hisham Alkuraya, Christopher Chung, Hassan Al-Dhibi, Nicola G. Ghazi, Thomas J. Conlon, Huimin Cai, Sanford L. Boye, Dilek Colak, Sawsan R. Nowilaty, Abdulrahman Al-Maghamsi, Wen-Tao Deng, Abdulrahman Alhommadi, Kang Zhang, Emad B. Abboud
Publikováno v:
Human Genetics. 135:327-343
MERTK is an essential component of the signaling network that controls phagocytosis in retinal pigment epithelium (RPE), the loss of which results in photoreceptor degeneration. Previous proof-of-concept studies have demonstrated the efficacy of gene
Autor:
Saleem Islam, Cristina Liberati, Roland W. Herzog, Shelley Collins, Lee Ann Lawson, Ibrahim Sacit Tuna, Barry J. Byrne, David D. Fuller, Kirsten E. Coleman, Manuela Corti, Thomas J. Conlon, Barbara K. Smith
Publikováno v:
Human gene therapy. Clinical development. 28(4)
A first-in-human trial of diaphragmatic gene therapy (AAV1-CMV-GAA) to treat respiratory and neural dysfunction in early-onset Pompe disease was conducted. The primary objective of this study was to assess the safety of rAAV1-CMV-hGAA vector delivere
Autor:
Kirsten E. Erger, Alok K. Sharma, Thomas J. Conlon, Jeffrey D. Chulay, Guo-jie Ye, David R. Knop, Peter Sonnentag, Kellie Howard
Publikováno v:
Human Gene Therapy Clinical Development. 26:177-184
Applied Genetic Technologies Corporation is developing a recombinant adeno-associated virus (rAAV) vector for treatment of X-linked retinoschisis (XLRS), an inherited retinal disease characterized by splitting (schisis) of the layers of the retina, w