Protein replacement therapy

Protein replacement therapy is a medical treatment that supplements or replaces a protein in patients in whom that particular protein is deficient or absent.^[1][2] There have been significant advances in this treatment. PRT is being tested in clinical trials with the diseases progeria and epidermolysis bullosa dystrophica as a potential treatment. For patients with epidermolysis bullosa dystrophica there have been promising results. ^[3][4]

See also

• Enzyme replacement therapy
• Gene therapy

References

1. Gorzelany JA, de Souza MP (March 2013). "Protein replacement therapies for rare diseases: a breeze for regulatory approval?". Science Translational Medicine. 5 (178): 178fs10. doi:10.1126/scitranslmed.3005007. PMID 23536010.
2. Crunkhorn S (June 2013). "Regulatory watch: enhanced chance of success for protein replacement therapies". Nature Reviews. Drug Discovery. 12 (6): 414. doi:10.1038/nrd4027. PMID 23722335. S2CID 30803636.
3. Labbe C (2014-02-05). "Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder". National Institute of Arthritis and Musculoskeletal and Skin Diseases. U.S. Department of Health and Human Services. Retrieved 2018-10-14.
4. Wang X, Ghasri P, Amir M, Hwang B, Hou Y, Khalili M, Khilili M, Lin A, Keene D, Uitto J, Woodley DT, Chen M (July 2013). "Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure". Molecular Therapy. 21 (7): 1335–44. doi:10.1038/mt.2013.87. PMC 3704128. PMID 23670575.

Further reading

• Stegmann TJ (December 2006). "Protein Therapy vs. Gene Therapy". DDNEWS.