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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. 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.

References

References

  1. (March 2013). "Protein replacement therapies for rare diseases: a breeze for regulatory approval?". Science Translational Medicine.
  2. (June 2013). "Regulatory watch: enhanced chance of success for protein replacement therapies". Nature Reviews. Drug Discovery.
  3. Labbe, Colleen. (2014-02-05). "Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder". U.S. Department of Health and Human Services.
  4. (July 2013). "Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure". Molecular Therapy.
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