Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial-Reference-Cited by-同舟云学术

Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial

Published:2019-09-01 Issue:9 Volume:30 Page:1067-1078
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Di Wei-Li¹,Lwin Su M.²,Petrova Anastasia¹,Bernadis Catina³,Syed Farhatullah¹,Farzaneh Farzin⁴,Moulding Dale⁵,Martinez Anna E.⁶,Sebire Neil J.⁷,Rampling Dyanne⁷,Virasami Alex⁷,Zamiri Mozheh⁸,Wang Wei⁹¹⁰,Hara Havinder¹,Kadiyirire Tendai²,Abdul-Wahab Alya²,Martinez-Queipo Magdalena²,Harper John I.¹,McGrath John A.²,Thrasher Adrian J.¹,Mellerio Jemima E.²,Qasim Waseem¹

Affiliation:

1. Infection, Immunity and Inflammation Programme, UCL GOS Institute of Child Health, London, United Kingdom

2. St. John's Institute of Dermatology, King's College London, Guy's Hospital, London, United Kingdom

3. Plastic Surgery Unit, Guy's and St. Thomas' NHS Foundation Trust, London, United Kingdom

4. Cell and Gene Therapy—King's (CGT-K), School of Cancer and Pharmaceutical Sciences, King's College London, London, United Kingdom

5. Light Microscopy Core Facility, UCL GOS Institute of Child Health, London, United Kingdom

6. Dermatology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

7. Histopathology Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom

8. School of Life Sciences, University of Dundee, Scotland, United Kingdom

9. Department of Translational Oncology, German Cancer Research Center, National Center for Tumor Diseases, Heidelberg, Heidelberg, Germany

10. GeneWerk GmbH, Heidelberg, Germany.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.049

Reference36 articles.

1. Mutations in SPINK5, encoding a serine protease inhibitor, cause Netherton syndrome

2. Netherton Syndrome

3. LEKTI proteolytic processing in human primary keratinocytes, tissue distribution and defective expression in Netherton syndrome

4. hK5 and hK7, two serine proteinases abundant in human skin, are inhibited by LEKTI domain 6

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