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Gene therapy and the skin
Author(s) -
Hengge U.R.,
Bardenheuer W.
Publication year - 2004
Publication title -
american journal of medical genetics part c: seminars in medical genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.419
H-Index - 101
eISSN - 1552-4876
pISSN - 1552-4868
DOI - 10.1002/ajmg.c.30038
Subject(s) - junctional epidermolysis bullosa (veterinary medicine) , xeroderma pigmentosum , genetic enhancement , gene , viral vector , epidermolysis bullosa , biology , genome editing , immune system , clinical trial , medicine , bioinformatics , computational biology , genetics , genome , phenotype , dna repair , recombinant dna
Significant progress has been made during the past decade in corrective gene therapy of the skin. This includes advances in vector technology, targeted gene expression, gene replacement, gene correction, and the availability of appropriate animal models for a variety of candidate diseases. While non‐viral integration of large genes such as essential basement membrane proteins has been mastered, new challenges such as the control of immune responses lie ahead of the research community. Among the first skin diseases, patients with junctional epidermolysis bullosa (JEB) and xeroderma pigmentosum (XP) will enter clinical trials. © 2004 Wiley‐Liss, Inc.