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Therapeutics development for cystic fibrosis: a successful model for a multisystem genetic disease.

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

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

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

Leprosy as a genetic disease

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

[BOOK] The history of a genetic disease: Duchenne muscular dystrophy or Meryon's disease

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

Causal Models for Investigating Complex Genetic Disease: II. What Causal Models Can Tell Us about Penetrance for Additive, Heterogeneity, and Multiplicative Two- …

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

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