In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
About this item
Full title
Author / Creator
Gurevich, Irina , Agarwal, Pooja , Zhang, PeiPei , Dolorito, John A. , Oliver, Stacie , Liu, Henry , Reitze, Nicholas , Sarma, Nikhil , Bagci, Isin Sinem , Sridhar, Kunju , Kakarla, Visesha , Yenamandra, Vamsi K. , O’Malley, Mark , Prisco, Marco , Tufa, Sara F. , Keene, Douglas R. , South, Andrew P. , Krishnan, Suma M. and Marinkovich, M. Peter
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
Recessive dystrophic epidermolysis bullosa (RDEB) is a lifelong genodermatosis associated with blistering, wounding, and scarring caused by mutations in
COL7A1
, the gene encoding the anchoring fibril component, collagen VII (C7). Here, we evaluated beremagene geperpavec (B-VEC), an engineered, non-replicating
COL7A1
containing herpes s...
Alternative Titles
Full title
In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
Authors, Artists and Contributors
Author / Creator
Agarwal, Pooja
Zhang, PeiPei
Dolorito, John A.
Oliver, Stacie
Liu, Henry
Reitze, Nicholas
Sarma, Nikhil
Bagci, Isin Sinem
Sridhar, Kunju
Kakarla, Visesha
Yenamandra, Vamsi K.
O’Malley, Mark
Prisco, Marco
Tufa, Sara F.
Keene, Douglas R.
South, Andrew P.
Krishnan, Suma M.
Marinkovich, M. Peter
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9018416
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_9018416
Other Identifiers
ISSN
1078-8956
E-ISSN
1546-170X
DOI
10.1038/s41591-022-01737-y
