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Literature DB >> 32283956

Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy.

Abstract

Shortly after the cystic fibrosis (CF) gene was identified in 1989, the race began to develop a gene therapy for this condition. Major efforts utilized full-length cystic fibrosis transmembrane conductance regulator packaged into adenovirus, adeno-associated virus (AAV), or liposomes and delivered to the airways. The drive to find a treatment for CF based on gene therapy drove the early stages of gene therapy in general, particularly those involving AAV gene therapy. Since general overviews of CF gene therapy have already been published, this review considers specifically the efforts using AAV and is focused on honoring the contributions of Dr. Barrie Carter.

Entities: Disease Species

Keywords: adeno-associated virus; clinical trials; cystic fibrosis; gene therapy; preclinical testing

Year: 2020 PMID： 32283956 PMCID： PMC7232693 DOI： 10.1089/hum.2020.046

Source DB: PubMed Journal: Hum Gene Ther ISSN： 1043-0342 Impact factor: 5.695

38 in total

1. Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector.

Authors: Jeffrey Sirninger; Christian Muller; Sofia Braag; Qiushi Tang; Hungwen Yue; Carol Detrisac; Thomas Ferkol; William B Guggino; Terence R Flotte
Journal: Hum Gene Ther Date: 2004-09 Impact factor: 5.695

2. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue.

Authors: D Duan; P Sharma; J Yang; Y Yue; L Dudus; Y Zhang; K J Fisher; J F Engelhardt
Journal: J Virol Date: 1998-11 Impact factor: 5.103

3. Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis.

Authors: B C Trapnell; C S Chu; P K Paakko; T C Banks; K Yoshimura; V J Ferrans; M S Chernick; R G Crystal
Journal: Proc Natl Acad Sci U S A Date: 1991-08-01 Impact factor: 11.205

4. Adeno-associated virus rep protein synthesis during productive infection.

Authors: B E Redemann; E Mendelson; B J Carter
Journal: J Virol Date: 1989-02 Impact factor: 5.103

5. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

Authors: John A Wagner; Ilynn B Nepomuceno; Anna H Messner; Mary Lynn Moran; Eric P Batson; Sue Dimiceli; Byron W Brown; Julie K Desch; Alexander M Norbash; Carol K Conrad; William B Guggino; Terence R Flotte; Jeffrey J Wine; Barrie J Carter; Thomas C Reynolds; Richard B Moss; Phyllis Gardner
Journal: Hum Gene Ther Date: 2002-07-20 Impact factor: 5.695

6. Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR.

Authors: M Egan; T Flotte; S Afione; R Solow; P L Zeitlin; B J Carter; W B Guggino
Journal: Nature Date: 1992-08-13 Impact factor: 49.962

Review 7. Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

Authors: Federico Mingozzi; Katherine A High
Journal: Blood Date: 2013-04-17 Impact factor: 22.113

8. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial.

Authors: Richard B Moss; David Rodman; L Terry Spencer; Moira L Aitken; Pamela L Zeitlin; David Waltz; Carlos Milla; Alan S Brody; John P Clancy; Bonnie Ramsey; Nicole Hamblett; Alison E Heald
Journal: Chest Date: 2004-02 Impact factor: 9.410

Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy.

1. Functional characterization of a recombinant adeno-associated virus 5-pseudotyped cystic fibrosis transmembrane conductance regulator vector.

2. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue.

3. Expression of the cystic fibrosis transmembrane conductance regulator gene in the respiratory tract of normal individuals and individuals with cystic fibrosis.

4. Adeno-associated virus rep protein synthesis during productive infection.

5. A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

6. Defective regulation of outwardly rectifying Cl- channels by protein kinase A corrected by insertion of CFTR.

Review 7. Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

8. Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial.

9. Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.

Review 10. Adeno-associated virus vectors for gene therapy.

1. Anticipating New Treatments for Cystic Fibrosis: A Global Survey of Researchers.

2. The use of adenoviral vectors in gene therapy and vaccine approaches.

3. In vivo Dominant-Negative Effect of an SCN5A Brugada Syndrome Variant.