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. Archived from the original on October 4, 2017. 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.