Repairing DNA damage in xeroderma pigmentosum: T4N5 lotion and gene therapy.
Patients with xeroderma pigmentosum (XP) have defective DNA repair and are at a high risk for cutaneous malignancies. Standard treatments for XP are limited in scope and effectiveness. Understanding the molecular etiology of XP has led to the development of novel therapeutic approaches, including enzyme and gene therapies. One new topical treatment utilizing bacteriophage T4 endonuclease 5 (T4N5) in a liposomal lotion is currently in clinical trials and has received a Fast Track designation from the FDA. Gene therapy for XP, while making leaps in preclinical studies, has been slower to develop due to tactical hurdles, but seems to have much potential for future treatment. If these treatments prove effective in lowering the risk of cancer in patients with XP, they may also be found useful in reducing skin cancers in other at-risk patient populations.
Zahid
Sarwar
S
Dermatology Service, Memorial Sloan-Kettering Cancer Center.
Brownell
Isaac
I
D000287:Administration, Topical; D000818:Animals; D004249:DNA Damage; D004260:DNA Repair; D004255:Deoxyribodipyrimidine Photo-Lyase / Q000008:administration & dosage / Q000627:therapeutic use; D043224:Deoxyribonuclease (Pyrimidine Dimer) / Q000008:administration & dosage / Q000627:therapeutic use; D015316:Genetic Therapy; D006801:Humans; D066298:In Vitro Techniques; D008081:Liposomes; D051379:Mice; D012878:Skin Neoplasms / Q000517:prevention & control; D014764:Viral Proteins / Q000008:administration & dosage / Q000627:therapeutic use; D014983:Xeroderma Pigmentosum / Q000235:genetics / Q000628:therapy
D008081:Liposomes; D014764:Viral Proteins; C470698:endonuclease V, phage T4; D043224:Deoxyribonuclease (Pyrimidine Dimer); D004255:Deoxyribodipyrimidine Photo-Lyase