A patient and family perspective on gene therapy for rare diseases.
The authors describe the view of patients and patient organisations on gene therapy research and gene therapy regulations. In particular, the added value of partnership between scientists and patient organisations, and patient involvement in the gene therapy field, are addressed.
Kent
Alastair
A
Genetic Interest Group (GIG), UK. egan@egan.eu
Oosterwijk
Cor
C
D002986:Clinical Trials as Topic; D005190:Family; D015316:Genetic Therapy / Q000379:methods / Q000639:trends*; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D006801:Humans; D035583:Rare Diseases / Q000235:genetics / Q000628:therapy*