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Protein replacement therapy

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27:
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
164: 191:"Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure" 189:
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).
36: 32: 264: 48: 243: 8: 215: 190: 145: 220: 137: 96: 20: 149: 75:"Protein replacement therapies for rare diseases: a breeze for regulatory approval?" 210: 202: 127: 86: 91: 74: 116:"Regulatory watch: enhanced chance of success for protein replacement therapies" 258: 224: 141: 100: 53: 165:"Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder" 206: 169:
National Institute of Arthritis and Musculoskeletal and Skin Diseases
132: 115: 28: 24: 188: 72: 256: 171:. U.S. Department of Health and Human Services 35:as a potential treatment. For patients with 107: 66: 214: 131: 113: 90: 241: 73:Gorzelany JA, de Souza MP (March 2013). 257: 162: 39:there have been promising results. 13: 244:"Protein Therapy vs. Gene Therapy" 235: 14: 276: 37:epidermolysis bullosa dystrophica 33:epidermolysis bullosa dystrophica 23:that supplements or replaces a 182: 156: 120:Nature Reviews. Drug Discovery 79:Science Translational Medicine 1: 242:Stegmann TJ (December 2006). 59: 92:10.1126/scitranslmed.3005007 7: 42: 17:Protein replacement therapy 10: 281: 49:Enzyme replacement therapy 114:Crunkhorn S (June 2013). 163:Labbe C (2014-02-05). 265:Medical treatments 207:10.1038/mt.2013.87 195:Molecular Therapy 21:medical treatment 272: 251: 229: 228: 218: 186: 180: 179: 177: 176: 160: 154: 153: 135: 111: 105: 104: 94: 85:(178): 178fs10. 70: 280: 279: 275: 274: 273: 271: 270: 269: 255: 254: 238: 236:Further reading 233: 232: 187: 183: 174: 172: 161: 157: 133:10.1038/nrd4027 112: 108: 71: 67: 62: 45: 12: 11: 5: 278: 268: 267: 253: 252: 237: 234: 231: 230: 201:(7): 1335–44. 181: 155: 106: 64: 63: 61: 58: 57: 56: 51: 44: 41: 9: 6: 4: 3: 2: 277: 266: 263: 262: 260: 249: 245: 240: 239: 226: 222: 217: 212: 208: 204: 200: 196: 192: 185: 170: 166: 159: 151: 147: 143: 139: 134: 129: 125: 121: 117: 110: 102: 98: 93: 88: 84: 80: 76: 69: 65: 55: 52: 50: 47: 46: 40: 38: 34: 30: 26: 22: 18: 247: 198: 194: 184: 173:. Retrieved 168: 158: 123: 119: 109: 82: 78: 68: 54:Gene therapy 16: 15: 175:2018-10-14 126:(6): 414. 60:References 259:Category 225:23670575 150:30803636 142:23722335 101:23536010 43:See also 29:progeria 216:3704128 25:protein 248:DDNEWS 223:  213:  148:  140:  99:  146:S2CID 19:is a 221:PMID 138:PMID 97:PMID 31:and 211:PMC 203:doi 128:doi 87:doi 261:: 246:. 219:. 209:. 199:21 197:. 193:. 167:. 144:. 136:. 124:12 122:. 118:. 95:. 81:. 77:. 250:. 227:. 205:: 178:. 152:. 130:: 103:. 89:: 83:5

Index

medical treatment
protein
progeria
epidermolysis bullosa dystrophica
epidermolysis bullosa dystrophica
Enzyme replacement therapy
Gene therapy
"Protein replacement therapies for rare diseases: a breeze for regulatory approval?"
doi
10.1126/scitranslmed.3005007
PMID
23536010
"Regulatory watch: enhanced chance of success for protein replacement therapies"
doi
10.1038/nrd4027
PMID
23722335
S2CID
30803636
"Protein Replacement Therapy Shows Promise in Treating Rare Skin Disorder"
"Topical application of recombinant type VII collagen incorporates into the dermal-epidermal junction and promotes wound closure"
doi
10.1038/mt.2013.87
PMC
3704128
PMID
23670575
"Protein Therapy vs. Gene Therapy"
Category
Medical treatments

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