Protein replacement therapy explained

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

Further reading

• Thomas J. . Stegmann . vanc . Protein Therapy vs. Gene Therapy . DDNEWS . December 2006 .

Notes and References

1. Gorzelany JA, de Souza MP . Protein replacement therapies for rare diseases: a breeze for regulatory approval? . Science Translational Medicine . 5 . 178 . 178fs10 . March 2013 . 23536010 . 10.1126/scitranslmed.3005007 . free .
2. Crunkhorn S . Regulatory watch: enhanced chance of success for protein replacement therapies . Nature Reviews. Drug Discovery . 12 . 6 . 414 . June 2013 . 23722335 . 10.1038/nrd4027 . 30803636 . free .
3. News: Colleen . Labbe . vanc . Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder. 2014-02-05. 2018-10-14 . National Institute of Arthritis and Musculoskeletal and Skin Diseases . U.S. Department of Health and Human Services .
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 . Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure . Molecular Therapy . 21 . 7 . 1335–44 . July 2013 . 23670575 . 3704128 . 10.1038/mt.2013.87 .