Gene therapy for the respiratory manifestations of cystic fibrosis
A MASTRANGELI, J HAY, SL BRODY… - Am J Respir Crit Care …, 1995 - atsjournals.org
Cystic fibrosis (CF) is caused by mutations of the cystic fibrosis transmembrane conductance
regulator (CFTR) gene. The major manifestations are on the airway epithelial surface, with
purulent mucus, recurrent infections, chronic inflammation, and loss of lung function.
Consequent to mutations in both parental genes, airway epithelial cells have insufficient
CFTR function. Because this can be corrected in vitro by transfer of the normal CFTR gene
into airway epithelial cells, it is reasonable to hypothesize that the respiratory manifestations …
regulator (CFTR) gene. The major manifestations are on the airway epithelial surface, with
purulent mucus, recurrent infections, chronic inflammation, and loss of lung function.
Consequent to mutations in both parental genes, airway epithelial cells have insufficient
CFTR function. Because this can be corrected in vitro by transfer of the normal CFTR gene
into airway epithelial cells, it is reasonable to hypothesize that the respiratory manifestations …
Evaluation of the Respiratory Epithelium of Normals and Individuals with Cystic Fibrosis for the Presence of Adenovirus E1a Sequences Relevant to the Use of E1a– …
NT Eissa, CS Chu, C Danel, RG Crystal - Human Gene Therapy, 1994 - liebertpub.com
Lung disease associated with disorders such as cystic fibrosis (CF) may be amenable to
somatic gene therapy in which there is delivery of the normal gene directly to the respiratory
epithelium using E1a–adenovirus (Ad) type 2-or 5-based vectors. For safety reasons, the Ad
vectors are rendered replication deficient by deletion of the E1a region. Because there is the
theoretical possibility of an E1a–replication-deficient vector replicating as a result of
recombination or complementation with Ad 2/5 E1a sequences present in the target cell, this …
somatic gene therapy in which there is delivery of the normal gene directly to the respiratory
epithelium using E1a–adenovirus (Ad) type 2-or 5-based vectors. For safety reasons, the Ad
vectors are rendered replication deficient by deletion of the E1a region. Because there is the
theoretical possibility of an E1a–replication-deficient vector replicating as a result of
recombination or complementation with Ad 2/5 E1a sequences present in the target cell, this …