Drugs / autologous skin sheet graft
last change Apr 2018 re-read 3 minutes ago

autologous skin sheet graft

targets SPINK5 via restoration

Developed for
Netherton syndrome
Investigated by
Great Ormond Street Hospital for Children NHS Foundation Trust

Trials 1

20142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT01545323 Apr 2014 → Apr 2018 Netherton syndrome Great Ormond Street Hospital for Children NHS Foundation Trust Unknown No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 2 sources stand behind the page.

FieldValueCited text
Known as autologous skin sheet graft ClinicalTrials.gov intervention name — accepted as the source's own label NCT01545323 ↗
Action Restore “The investigators have used a disabled virus (vector) to carry a functional copy of the SPINK5 gene into skin stem cells.” NCT01545323 ↗
Route Other “autologous epidermal sheets generated from ex vivo gene-corrected keratinocyte stem cells, which will be grafted onto patients with mutation-proven NS” PMID 24329107 ↗ Dec 2013
Target SPINK5 “A SIN-lentiviral vector encoding the codon-optimized SPINK5 gene under the control of a 572 bp element derived from the human involucrin promoter can confer...” PMID 24329107 ↗ Dec 2013