Systemic Protein Therapy for Recessive Dystrophic Epidermolysis Bullosa: How Far Are We from Clinical Translation?

被引:11
作者
Hovnanian, Alain [1 ,2 ,3 ]
机构
[1] Univ Paris Descartes Sorbonne Paris Cite, Imagine Inst, Paris, France
[2] INSERM, U781, Paris, France
[3] Hop Necker Enfants Malad, Dept Genet, Paris, France
关键词
VII COLLAGEN; GENE-THERAPY; TRANSPLANTATION; INJECTION;
D O I
10.1038/jid.2013.137
中图分类号
R75 [皮肤病学与性病学];
学科分类号
100206 ;
摘要
In this issue, Wood ley et al. report restoration of anchoring fibril formation and dermal-epidermal adherence in a murine model of recessive dystrophic epidermolysis bullosa (RDEB) by intravenous injection of recombinant human type VII collagen. This work follows a previous report by the same group of the surprising capability of intradermally injected type VII collagen protein to reverse RDEB, and it opens new therapeutic avenues.
引用
收藏
页码:1719 / 1721
页数:3
相关论文
共 13 条
  • [1] Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges
    Desnick, R. J.
    Schuchman, E. H.
    [J]. ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 13, 2012, 13 : 307 - 335
  • [2] The classification of inherited epidermolysis bullosa (EB): Report of the Third International Consensus Meeting on Diagnosis and Classification of EB
    Fine, Jo-David
    Eady, Robin A. J.
    Bauer, Eugene A.
    Bauer, Johann W.
    Bruckner-Tuderman, Leena
    Heagerty, Adrian
    Hintner, Helmut
    Hovnanian, Alain
    Jonkman, Marcel E.
    Leigh, Irene
    McGrath, John A.
    Mellerio, Jemima E.
    Murrell, Dedee E.
    Shimizu, Hiroshi
    Uitto, Jouni
    Vahlquist, Anders
    Woodley, David
    Zambruno, Giovanna
    [J]. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY, 2008, 58 (06) : 931 - 950
  • [3] Protein therapeutics for junctional epidermolysis bullosa:: Incorporation of recombinant β3 chain into laminin 332 in β3-/- keratinocytes in vitro
    Igoucheva, Olga
    Kelly, Aislinn
    Uitto, Jouni
    Alexeev, Vitali
    [J]. JOURNAL OF INVESTIGATIVE DERMATOLOGY, 2008, 128 (06) : 1476 - 1486
  • [4] Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells
    Mavilio, Fulvio
    Pellegrini, Graziella
    Ferrari, Stefano
    Di Nunzio, Francesca
    Di Iorio, Enzo
    Recchia, Alessandra
    Maruggi, Giulietta
    Ferrari, Giuliana
    Provasi, Elena
    Bonini, Chiara
    Capurro, Sergio
    Conti, Andrea
    Magnoni, Cristina
    Giannetti, Alberto
    De Luca, Michele
    [J]. NATURE MEDICINE, 2006, 12 (12) : 1397 - 1402
  • [5] Immune reactivity to type VII collagen: implications for gene therapy of recessive dystrophic epidermolysis bullosa
    Pendaries, V.
    Gasc, G.
    Titeux, M.
    Leroux, C.
    Vitezica, Z. G.
    Mejia, J. E.
    Decha, A.
    Loiseau, P.
    Bodemer, C.
    Prost-Squarcioni, C.
    Hovnanian, A.
    [J]. GENE THERAPY, 2010, 17 (07) : 930 - 937
  • [6] Injection of Recombinant Human Type VII Collagen Corrects the Disease Phenotype in a Murine Model of Dystrophic Epidermolysis Bullosa
    Remington, Jennifer
    Wang, Xinyi
    Hou, Yingpin
    Zhou, Hui
    Burnett, Julie
    Muirhead, Trevor
    Uitto, Jouni
    Keene, Douglas R.
    Woodley, David T.
    Chen, Mei
    [J]. MOLECULAR THERAPY, 2009, 17 (01) : 26 - 33
  • [7] Long-Term Type VII Collagen Restoration to Human Epidermolysis Bullosa Skin Tissue
    Siprashvili, Zurab
    Nguyen, Ngon T.
    Bezchinsky, Maria Y.
    Marinkovich, M. Peter
    Lane, Alfred T.
    Khavari, Paul A.
    [J]. HUMAN GENE THERAPY, 2010, 21 (10) : 1299 - 1310
  • [8] SIN Retroviral Vectors Expressing COL7A1 Under Human Promoters for Ex Vivo Gene Therapy of Recessive Dystrophic Epidermolysis Bullosa
    Titeux, Matthias
    Pendaries, Valerie
    Zanta-Boussif, Maria A.
    Decha, Audrey
    Pironon, Nathalie
    Tonasso, Laure
    Mejia, Jose E.
    Brice, Agnes
    Danos, Olivier
    Hovnanian, Alain
    [J]. MOLECULAR THERAPY, 2010, 18 (08) : 1509 - 1518
  • [9] Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa
    Wagner, John E.
    Ishida-Yamamoto, Akemi
    McGrath, John A.
    Hordinsky, Maria
    Keene, Douglas R.
    Riddle, Megan J.
    Osborn, Mark J.
    Lund, Troy
    Dolan, Michelle
    Blazar, Bruce R.
    Tolar, Jakub
    [J]. NEW ENGLAND JOURNAL OF MEDICINE, 2010, 363 (07) : 629 - 639
  • [10] Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa
    Wong, Tracy
    Gammon, Luke
    Liu, Lu
    Mellerio, Jemima E.
    Dopping-Hepenstal, Patricia J. C.
    Pacy, John
    Elia, George
    Jeffery, Rosemary
    Leigh, Irene M.
    Navsaria, Harshad
    McGrath, John A.
    [J]. JOURNAL OF INVESTIGATIVE DERMATOLOGY, 2008, 128 (09) : 2179 - 2189