Medicine:Protein replacement therapy

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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

References

  1. "Protein replacement therapies for rare diseases: a breeze for regulatory approval?". Science Translational Medicine 5 (178): 178fs10. March 2013. doi:10.1126/scitranslmed.3005007. PMID 23536010. 
  2. "Regulatory watch: enhanced chance of success for protein replacement therapies". Nature Reviews. Drug Discovery 12 (6): 414. June 2013. doi:10.1038/nrd4027. PMID 23722335. 
  3. Labbe, Colleen (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). https://www.niams.nih.gov/newsroom/spotlight-on-research/protein-replacement-therapy-shows-promise. 
  4. "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. doi:10.1038/mt.2013.87. PMID 23670575. 

Further reading