Phase I Study Protocol for Ex Vivo Lentiviral Gene Therapy for the Inherited Skin Disease, Netherton Syndrome-Reference-Cited by-同舟云学术

Phase I Study Protocol for Ex Vivo Lentiviral Gene Therapy for the Inherited Skin Disease, Netherton Syndrome

Published:2013-12 Issue:4 Volume:24 Page:182-190
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Di Wei-Li¹,Mellerio Jemima E.²,Bernadis Catina³,Harper John¹,Abdul-Wahab Alya²,Ghani Sumera¹,Chan Lucas⁴,Martinez-Queipo Magdalena²,Hara Havinder¹,McNicol Anne-Marie¹,Farzaneh Farzin⁴,McGrath John²,Thrasher Adrian¹,Qasim Waseem¹

Affiliation:

1. UCL Institute of Child Health & Great Ormond Street Hospital, London WC1N 1EH, United Kingdom.

2. St. John's Institute of Dermatology, King's College London (Guy's Campus), London SE1 9RT, United Kingdom.

3. Plastic Surgery Unit, Guy's and St. Thomas' National Health Service Foundation Trust, London SE1 7EH, United Kingdom.

4. Rayne Cell Therapy Suite, Department of Haematological Medicine, King's College London, London SE5 9NU, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2013.195

Reference37 articles.

1. Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

2. Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

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

4. Elastase 2 is expressed in human and mouse epidermis and impairs skin barrier function in Netherton syndrome through filaggrin and lipid misprocessing

5. Kallikrein 5 induces atopic dermatitis–like lesions through PAR2-mediated thymic stromal lymphopoietin expression in Netherton syndrome

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