MA Ashlock… - Annual review of medicine, 2011 - ncbi.nlm.nih.gov Cystic fibrosis (CF) is a progressive genetic disease primarily involving the respiratory and gastrointestinal tracts. Multiple therapies directed at CF symptoms and clinical management strategies have emerged from iterative cycles of therapeutics development, helping to change the face of ... Cited by 1 - Related articles
F Mingozzi… - Nature Reviews Genetics, 2011 - nature.com In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively used for this purpose and have shown therapeutic efficacy in a range of animal models. ... Cited by 8 - Related articles - All 3 versions
A Alter, A Grant, L Abel, A Alcaïs… - Mammalian Genome, 2011 - Springer Abstract Leprosy (Hansen's disease) is a human infectious disease whose etiological agent, Mycobacterium leprae, was identified by GHA Hansen in the 19th century. Despite the high efficacy of multidrug therapy (\0.1% annual relapse rate), transmission is persistent. In 2008, ... Cited by 2 - Related articles - All 5 versions
AEH Emery… - 2011 - books.google.com 1 Great Clarendon Street, Oxford OX2 6DP Oxford University Press is a department of the University of Oxford. It furthers the University's objective of excellence in research, scholarship, and education by publishing worldwide in Oxford New York Auckland Cape Town Dar es Salaam Hong ... Cited by 26 - Related articles - All 2 versions
AM Madsen, R Ottman… - Human Heredity, 2011 - content.karger.com Background/Aims: Statistical geneticists commonly use certain two-locus penetrance models because these models are familiar and mathematically tractable. We investigate whether and under what circumstances these two-locus penetrance models correspond to models of ... Cited by 1