title: Enzyme therapy of xeroderma pigmentosum: safety and efficacy testing of T4N5 liposome lotion containing a prokaryotic DNA repair enzyme.
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.
authors.affiliation: Applied Genetics Inc., Freeport, New York 11520, USA.
authors.forename: D
authors.initials: D
authors.lastname: Yarosh
authors.forename: J
authors.initials: J
authors.lastname: Klein
authors.forename: J
authors.initials: J
authors.lastname: Kibitel
authors.forename: L
authors.initials: L
authors.lastname: Alas
authors.forename: A
authors.initials: A
authors.lastname: O'Connor
authors.forename: B
authors.initials: B
authors.lastname: Cummings
authors.forename: D
authors.initials: D
authors.lastname: Grob
authors.forename: D
authors.initials: D
authors.lastname: Gerstein
authors.forename: B A
authors.initials: BA
authors.lastname: Gilchrest
authors.forename: M
authors.initials: M
authors.lastname: Ichihashi
authors.forename: M
authors.initials: M
authors.lastname: Ogoshi
authors.forename: M
authors.initials: M
authors.lastname: Ueda
authors.forename: V
authors.initials: V
authors.lastname: Fernandez
authors.forename: C
authors.initials: C
authors.lastname: Chadwick
authors.forename: C S
authors.initials: CS
authors.lastname: Potten
authors.forename: C M
authors.initials: CM
authors.lastname: Proby
authors.forename: A R
authors.initials: AR
authors.lastname: Young
authors.forename: J L
authors.initials: JL
authors.lastname: Hawk
mesh_terms: D000293:Adolescent; D000328:Adult; D000818:Animals; D002648:Child; D004260:DNA Repair; D043224:Deoxyribonuclease (Pyrimidine Dimer); D004337:Drug Carriers; D004706:Endodeoxyribonucleases / Q000008:administration & dosage* / Q000009:adverse effects / Q000633:toxicity; D005260:Female; D006801:Humans; D008081:Liposomes; D008297:Male; D051379:Mice; D009824:Ointments; D014983:Xeroderma Pigmentosum / Q000188:drug therapy* / Q000235:genetics
chemical_list: D004337:Drug Carriers; D008081:Liposomes; D009824:Ointments; D004706:Endodeoxyribonucleases; D043224:Deoxyribonuclease (Pyrimidine Dimer)