Literature DB >> 24692191

Gene therapy for skin diseases.

Emily Gorell1, Ngon Nguyen, Alfred Lane, Zurab Siprashvili.   

Abstract

The skin possesses qualities that make it desirable for gene therapy, and studies have focused on gene therapy for multiple cutaneous diseases. Gene therapy uses a vector to introduce genetic material into cells to alter gene expression, negating a pathological process. This can be accomplished with a variety of viral vectors or nonviral administrations. Although results are promising, there are several potential pitfalls that must be addressed to improve the safety profile to make gene therapy widely available clinically.

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Year:  2014        PMID: 24692191      PMCID: PMC3968787          DOI: 10.1101/cshperspect.a015149

Source DB:  PubMed          Journal:  Cold Spring Harb Perspect Med        ISSN: 2157-1422            Impact factor:   6.915


  112 in total

1.  Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa.

Authors:  Eva M Murauer; Yannick Gache; Iris K Gratz; Alfred Klausegger; Wolfgang Muss; Christina Gruber; Guerrino Meneguzzi; Helmut Hintner; Johann W Bauer
Journal:  J Invest Dermatol       Date:  2010-08-19       Impact factor: 8.551

2.  Cancer regression in patients after transfer of genetically engineered lymphocytes.

Authors:  Richard A Morgan; Mark E Dudley; John R Wunderlich; Marybeth S Hughes; James C Yang; Richard M Sherry; Richard E Royal; Suzanne L Topalian; Udai S Kammula; Nicholas P Restifo; Zhili Zheng; Azam Nahvi; Christiaan R de Vries; Linda J Rogers-Freezer; Sharon A Mavroukakis; Steven A Rosenberg
Journal:  Science       Date:  2006-08-31       Impact factor: 47.728

Review 3.  Retroviral vectors.

Authors:  A D Miller
Journal:  Curr Top Microbiol Immunol       Date:  1992       Impact factor: 4.291

4.  A new DNA vehicle for nonviral gene delivery: supercoiled minicircle.

Authors:  A M Darquet; B Cameron; P Wils; D Scherman; J Crouzet
Journal:  Gene Ther       Date:  1997-12       Impact factor: 5.250

Review 5.  The molecular genetics of basement membrane diseases.

Authors:  M P Marinkovich
Journal:  Arch Dermatol       Date:  1993-12

6.  TALEN-based gene correction for epidermolysis bullosa.

Authors:  Mark J Osborn; Colby G Starker; Amber N McElroy; Beau R Webber; Megan J Riddle; Lily Xia; Anthony P DeFeo; Richard Gabriel; Manfred Schmidt; Christof von Kalle; Daniel F Carlson; Morgan L Maeder; J Keith Joung; John E Wagner; Daniel F Voytas; Bruce R Blazar; Jakub Tolar
Journal:  Mol Ther       Date:  2013-04-02       Impact factor: 11.454

7.  Microneedle delivery of plasmid DNA to living human skin: Formulation coating, skin insertion and gene expression.

Authors:  Marc Pearton; Verena Saller; Sion A Coulman; Chris Gateley; Alexander V Anstey; Vladimir Zarnitsyn; James C Birchall
Journal:  J Control Release       Date:  2012-04-10       Impact factor: 9.776

8.  Peripheral blood lymphocytes genetically modified to express the self/tumor antigen MAGE-A3 induce antitumor immune responses in cancer patients.

Authors:  Raffaella Fontana; Marco Bregni; Arcadi Cipponi; Laura Raccosta; Cristina Rainelli; Daniela Maggioni; Francesca Lunghi; Fabio Ciceri; Sylvain Mukenge; Claudio Doglioni; Didier Colau; Pierre G Coulie; Claudio Bordignon; Catia Traversari; Vincenzo Russo
Journal:  Blood       Date:  2008-12-12       Impact factor: 22.113

9.  Cell suspension cultures of allogenic keratinocytes are efficient carriers for ex vivo gene transfer and accelerate the healing of full-thickness skin wounds by overexpression of human epidermal growth factor.

Authors:  Jan Jeroen Vranckx; Daniela Hoeller; Patrik E M Velander; Christoph F P Theopold; Nicola Petrie; Akira Takedo; Elof Eriksson; Feng Yao
Journal:  Wound Repair Regen       Date:  2007 Sep-Oct       Impact factor: 3.617

10.  Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone.

Authors:  David T Woodley; Gerald G Krueger; Cynthia M Jorgensen; Janet A Fairley; Tom Atha; Yi Huang; Lawrence Chan; Douglas R Keene; Mei Chen
Journal:  J Invest Dermatol       Date:  2003-11       Impact factor: 8.551

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  14 in total

1.  Gene Therapy for Epidermolysis Bullosa: Sticky Business.

Authors:  Alexander Nyström; Leena Bruckner-Tuderman
Journal:  Mol Ther       Date:  2016-12       Impact factor: 11.454

Review 2.  Nanomedicine and advanced technologies for burns: Preventing infection and facilitating wound healing.

Authors:  Mirza Ali Mofazzal Jahromi; Parham Sahandi Zangabad; Seyed Masoud Moosavi Basri; Keyvan Sahandi Zangabad; Ameneh Ghamarypour; Amir R Aref; Mahdi Karimi; Michael R Hamblin
Journal:  Adv Drug Deliv Rev       Date:  2017-08-04       Impact factor: 15.470

Review 3.  [Ex vivo stem cell gene therapy of the skin : Ready for clinical use?]

Authors:  Ulrich Koller
Journal:  Hautarzt       Date:  2020-02       Impact factor: 0.751

4.  An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness.

Authors:  Seoghyun Lee; Minho Won; Ran Hee Hwang; Gang Min Hur; Hyunju Ro
Journal:  J Vis Exp       Date:  2018-05-07       Impact factor: 1.355

Review 5.  New developments in the molecular treatment of ichthyosis: review of the literature.

Authors:  M D W Joosten; J M K Clabbers; N Jonca; J Mazereeuw-Hautier; A H Gostyński
Journal:  Orphanet J Rare Dis       Date:  2022-07-15       Impact factor: 4.303

6.  Selective Ablation of BCL11A in Epidermal Keratinocytes Alters Skin Homeostasis and Accelerates Excisional Wound Healing In Vivo.

Authors:  Nilika Bhattacharya; Arup K Indra; Gitali Ganguli-Indra
Journal:  Cells       Date:  2022-07-03       Impact factor: 7.666

Review 7.  Juvenile idiopathic arthritis in Harlequin ichthyosis, a rare combination or the clinical spectrum of the disease? Report of a child treated with etanercept and review of the literature.

Authors:  Francesco Baldo; Michela Brena; Simone Carbogno; Francesca Minoia; Stefano Lanni; Sophie Guez; Antonella Petaccia; Carlo Agostoni; Rolando Cimaz; Giovanni Filocamo
Journal:  Pediatr Rheumatol Online J       Date:  2021-06-03       Impact factor: 3.054

Review 8.  Pathogenetic Therapy of Epidermolysis Bullosa: Current State and Prospects.

Authors:  I I Ryumina; K V Goryunov; D N Silachev; Yu A Shevtsova; V A Babenko; N M Marycheva; Yu Yu Kotalevskaya; V V Zubkov; G T Zubkov
Journal:  Bull Exp Biol Med       Date:  2021-05-29       Impact factor: 0.804

Review 9.  Immunological challenges associated with artificial skin grafts: available solutions and stem cells in future design of synthetic skin.

Authors:  Saurabh Dixit; Dieudonné R Baganizi; Rajnish Sahu; Ejowke Dosunmu; Atul Chaudhari; Komal Vig; Shreekumar R Pillai; Shree R Singh; Vida A Dennis
Journal:  J Biol Eng       Date:  2017-12-13       Impact factor: 4.355

10.  Ecdysone Receptor-based Singular Gene Switches for Regulated Transgene Expression in Cells and Adult Rodent Tissues.

Authors:  Seoghyun Lee; Kyung-Cheol Sohn; Dae-Kyoung Choi; Minho Won; Kyeong Ah Park; Sung-Kyu Ju; Kidong Kang; Young-Ki Bae; Gang Min Hur; Hyunju Ro
Journal:  Mol Ther Nucleic Acids       Date:  2016-09-27       Impact factor: 10.183

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