Zobrazeno 1 - 5
of 5
pro vyhledávání: '"Laura Conatser"'
Autor:
Brian C. Gilger, Telmo Llanga, Kendall Carlin, Patricia O'Donnell, Keiko Miyadera, Matthew L. Hirsch, R. Jude Samulski, Joanne Kurtzberg, Liujiang Song, Jessica H. Bagel, Laura Conatser
Publikováno v:
Mol Ther
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disease characterized by severe phenotypes, including corneal clouding. MPS I is caused by mutations in alpha-l-iduronidase (IDUA), a ubiquitous enzyme that catalyzes th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::93abd572904d48271b0ce3491634c7a0
https://doi.org/10.1016/j.ymthe.2020.04.004
https://doi.org/10.1016/j.ymthe.2020.04.004
Autor:
Amanda Lee Dobbins, R. Jude Samulski, Laura Conatser, Victoria M. Lue, Chengwen Li, Lauriel F. Earley, Matthew L. Hirsch
Publikováno v:
Hum Gene Ther
Adeno-associated viral vectors have been successfully used in laboratory and clinical settings for efficient gene delivery. In these vectors, 96% of the adeno-associated virus (AAV) genome is replaced with a gene cassette of interest, leaving only th
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::33035f63b330c1d5478e5a3412bf73b7
https://pubmed.ncbi.nlm.nih.gov/31914802
https://pubmed.ncbi.nlm.nih.gov/31914802
Autor:
Matthew L. Hirsch, Violeta Zaric, Brian C. Gilger, Laura Conatser, Telmo Llanga, Liujiang Song
Publikováno v:
Gene Therapy. 25:402-414
AAV gene therapy approaches in the posterior eye resulted in the first FDA-approved gene therapy-based drug. However, application of AAV vectorology to the anterior eye has yet to enter even a Phase I trial. Furthermore, the simple and safe subconjun
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::12b522037c04af67c3c557c04b93f8f2
https://doi.org/10.1038/s41434-018-0035-6
https://doi.org/10.1038/s41434-018-0035-6
Autor:
Matthew L. Hirsch, Brian C. Gilger, Laura Conatser, Nicholas Buglak, Sara M. Smith, Jacklyn H. Salmon, Jerry Wu, Rich Davis
Publikováno v:
Scientific Reports, Vol 7, Iss 1, Pp 1-11 (2017)
Scientific Reports
Scientific Reports
Over 1.5 million individuals suffer from cornea vascularization due to genetic and/or environmental factors, compromising visual acuity and often resulting in blindness. Current treatments of corneal vascularization are limited in efficacy and elicit
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fcc3fba459d0fb9baff5b3876344d7d1
http://link.springer.com/article/10.1038/s41598-017-18002-9
http://link.springer.com/article/10.1038/s41598-017-18002-9
Autor:
Telmo Llanga, Nadia Nagy, Catherine F. Dial, R. Bryan Sutton, Matthew L. Hirsch, Laura Conatser
Publikováno v:
Molecular Therapy. 25:2150-2162
Dysferlinopathy is an autosomal recessive muscular dystrophy characterized by the progressive loss of motility that is caused by mutations throughout the DYSF gene. There are currently no approved therapies that ameliorate or reverse dysferlinopathy.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::723e9ab65d205a92004ba8c6df1930a8
https://doi.org/10.1016/j.ymthe.2017.05.013
https://doi.org/10.1016/j.ymthe.2017.05.013