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.
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Delivery of T4-pdg in mouse models increased the rate of CPD removal, reduced the frequency of SCC, and minimized UVB-induced immune suppression.
“Delivery of T4-pdg in mouse models increased the rate of CPD removal, reduced the frequency of SCC, and minimized UVB-induced immune suppression.”
From Modulation of UVB-induced Carcinogenesis by Activation of Alternative DNA Repair Pathways (2018) · cites this paper as [16]
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De Sanctis-Cacchione Syndrome with Subdural Effusion
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Coelho, A. A., Dias, E. P. S., & Kipnis, A. (2026) · Frontiers in Systems Biology · doi:10.3389/fsysb.2026.1686085
- Authors
- Yarosh, D., Klein, J., Kibitel, J., Alas, L., O'Connor, A., Cummings, B., Grab, D., Gerstein, D., Gilchrest, B. A., Ichihashi, M., Ogoshi, M., Ueda, M., Fernandez, V., Chadwick, C., Potten, C. S., Proby, C. M., Young, A. R., & Hawk, J. L. M.
- Affiliations
- Boston University (US)
- Kobe University Hospital (JP)
- Cancer Research UK Manchester Institute (GB)
- Corresponding author
- Yarosh, D.
- Venue
- Photodermatology, Photoimmunology & Photomedicine
- Publisher
- Wiley
- Published
- 1996-06
- Volume
- 12
- Issue
- 3
- Pages
- 122-130
- ISSN
- 0905-4383 (ISSN-L)
- 1600-0781
- OpenAlex
- W2005403770
- Cited by
- 81
- References
- 31
- Citations by year
- 2026: 2 · 2025: 1 · 2024: 3 · 2023: 2 · 2022: 1 · 2021: 5 · 2020: 1 · 2019: 3 · 2018: 3 · 2016: 3 · 2014: 2 · 2013: 4
- FWCI
- 1.01
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Photodermatology Photoimmunology & Photomedicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › DNA Repair Mechanisms
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetic and rare skin diseases.
- Health Sciences › Medicine › Dermatology › Skin Protection and Aging
- Keywords
- xeroderma pigmentosum, enzyme therapy, DNA repair enzymes, topical drug delivery, UV-induced DNA damage, cyclobutane pyrimidine dimers, erythema, safety testing, efficacy evaluation
- MeSH terms
- Adolescent, Adult, Animals, Child, DNA Repair, Drug Carriers, Endodeoxyribonucleases, Female, Humans, Liposomes, Male, Ointments, Xeroderma Pigmentosum, Deoxyribonuclease (Pyrimidine Dimer), Mice
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