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CAVEAT TO PROTEIN REPLACEMENT THERAPY FOR GENETIC DISEASE:IMMUNOLOGICAL IMPLICATIONS OF ACCURATE MOLECULAR DIAGNOSIS

Authors
Journal
The Lancet
0140-6736
Publisher
Elsevier
Publication Date
Volume
302
Issue
7830
Identifiers
DOI: 10.1016/s0140-6736(73)92489-6
Disciplines
  • Biology
  • Medicine

Abstract

Abstract Replacement therapy with normal protein is being tried more and more often in cases where the specific protein abnormality in a genetic disease has been identified. Results are sometimes dramatic, but mutants who produce none of the protein in question may treat the normal replacement protein as a foreign antigen and mount an immune response which could lead to treatment failure and iatrogenic disease. Where replacement therapy is contemplated the susceptibility of the mutant can be determined by searching for cross-reactive material (C.R.M.). C.R.M.-negative patients are more likely to respond unfavourably to replacement therapy than C.R.M.-positive (and C.R.M.- reduced) mutants.

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