Enzyme therapy of xeroderma pigmentosum: safety and efficacy testing of T4N5 liposome lotion containing a prokaryotic DNA repair enzyme

Photodermatol Photoimmunol Photomed. 1996 Jun;12(3):122-30. doi: 10.1111/j.1600-0781.1996.tb00188.x.

Abstract

Xeroderma pigmentosum (XP) is a rare genetic disease in which patients are defective in DNA repair and are extremely sensitive to solar UV radiation exposure. A new treatment approach was tested in these patients, in which a prokaryotic DNA repair enzyme specific for UV-induced DNA damage was delivered into the skin by means of topically applied liposomes to supplement the deficient activity. Acute and chronic safety testing in both mice and humans showed neither adverse reactions nor significant changes in serum chemistry or in skin histology. The skin of XP patients treated with the DNA repair liposomes had fewer cyclobutylpyrimidine dimers in DNA and showed less erythema than did control sites. The results encourage further clinical testing of this new enzyme therapy approach.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Animals
  • Child
  • DNA Repair
  • Deoxyribonuclease (Pyrimidine Dimer)
  • Drug Carriers
  • Endodeoxyribonucleases / administration & dosage*
  • Endodeoxyribonucleases / adverse effects
  • Endodeoxyribonucleases / toxicity
  • Female
  • Humans
  • Liposomes
  • Male
  • Mice
  • Ointments
  • Xeroderma Pigmentosum / drug therapy*
  • Xeroderma Pigmentosum / genetics

Substances

  • Drug Carriers
  • Liposomes
  • Ointments
  • Endodeoxyribonucleases
  • Deoxyribonuclease (Pyrimidine Dimer)