CFTR gene transduction in neonatal rabbits using an adeno-associated virus (AAV) vector.
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Rubenstein RC, et al. Among authors: flotte tr.
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PMID: 9274714
Patients with cystic fibrosis develop lung disease after birth, therefore CFTR gene replacement therapy should be most efficacious in the neonatal period prior to the onset of pulmonary damage. An adeno-associated virus (AAV) vector, SA306 (Flotte TR et al Proc Natl …
Patients with cystic fibrosis develop lung disease after birth, therefore CFTR gene replacement therapy should be most efficacious in the ne …