SlideShare a Scribd company logo
1 of 11
Gene Therapy for the Treatment of Cystic Fibrosis  Stephanie M. Delgado RISE Program BIOL 3095
Cystic Fibrosis Autosomal recessive genetic disorder CFTR gene
Gene Therapy What is gene therapy? Objective
Degree of correction required Minimal percentage of cells with a non-CF phenotyperequired to normalize the functions of the CF bronchial epithelial layer.
Barriers for gene delivery Defense mechanisms Delivery systems
Vectors Vectors ,[object Object]
Objective: Generation of a functional protein,[object Object]
Adeno-associated
Lentivirus
Poxvirus, Sendai, Herpes virus ,[object Object]
Less likely to induce immune response,[object Object]

More Related Content

What's hot (20)

Transfection method
Transfection methodTransfection method
Transfection method
 
Site directed mutagenesis
Site  directed mutagenesisSite  directed mutagenesis
Site directed mutagenesis
 
Whole genome sequencing
Whole genome sequencingWhole genome sequencing
Whole genome sequencing
 
ONCOGENE AND TUMOUR SUPPRESSOR GENE
ONCOGENE AND TUMOUR SUPPRESSOR GENEONCOGENE AND TUMOUR SUPPRESSOR GENE
ONCOGENE AND TUMOUR SUPPRESSOR GENE
 
Transfection
TransfectionTransfection
Transfection
 
Oncogenes
Oncogenes Oncogenes
Oncogenes
 
Baculovirus expression vector system
Baculovirus expression vector systemBaculovirus expression vector system
Baculovirus expression vector system
 
Retroviral Vectors
Retroviral VectorsRetroviral Vectors
Retroviral Vectors
 
Sanger sequencing (DNA sequencing by ENZYMATIC METHOD)
Sanger sequencing (DNA sequencing by ENZYMATIC METHOD)Sanger sequencing (DNA sequencing by ENZYMATIC METHOD)
Sanger sequencing (DNA sequencing by ENZYMATIC METHOD)
 
Microarray technology and applications
Microarray technology and applicationsMicroarray technology and applications
Microarray technology and applications
 
miRNA
miRNAmiRNA
miRNA
 
tumor suppressor gene, prb, p53
tumor suppressor gene, prb, p53tumor suppressor gene, prb, p53
tumor suppressor gene, prb, p53
 
Probe labeling
Probe labelingProbe labeling
Probe labeling
 
Gene knockout
Gene knockoutGene knockout
Gene knockout
 
Method of gene transfer
Method of gene transferMethod of gene transfer
Method of gene transfer
 
Growth factors
Growth factorsGrowth factors
Growth factors
 
VIRAL VECTORS FOR GENE TRANSFER
VIRAL VECTORS FOR GENE TRANSFERVIRAL VECTORS FOR GENE TRANSFER
VIRAL VECTORS FOR GENE TRANSFER
 
Next generation sequencing
Next generation sequencingNext generation sequencing
Next generation sequencing
 
P uc vectors
P uc vectorsP uc vectors
P uc vectors
 
Chromosome engineering
Chromosome engineeringChromosome engineering
Chromosome engineering
 

Viewers also liked

Viewers also liked (20)

Cystic fibrosis presentation
Cystic fibrosis presentationCystic fibrosis presentation
Cystic fibrosis presentation
 
Cystic fibrosis
Cystic fibrosisCystic fibrosis
Cystic fibrosis
 
Cystic Fibrosis Presentation
Cystic Fibrosis PresentationCystic Fibrosis Presentation
Cystic Fibrosis Presentation
 
Cystic fibrosis
Cystic fibrosisCystic fibrosis
Cystic fibrosis
 
CYSTIC FIBROSIS
CYSTIC FIBROSISCYSTIC FIBROSIS
CYSTIC FIBROSIS
 
Cystic Fibrosis
Cystic FibrosisCystic Fibrosis
Cystic Fibrosis
 
Gene therapy ppt
Gene therapy pptGene therapy ppt
Gene therapy ppt
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Cystic fibrosis powerpoint pict
Cystic fibrosis powerpoint pictCystic fibrosis powerpoint pict
Cystic fibrosis powerpoint pict
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Cystic fibrosis
Cystic fibrosisCystic fibrosis
Cystic fibrosis
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 
Cystic Fibrosis. Brief explanation
Cystic Fibrosis. Brief explanationCystic Fibrosis. Brief explanation
Cystic Fibrosis. Brief explanation
 
cystic fibrosis
cystic fibrosiscystic fibrosis
cystic fibrosis
 
channelopathies in Cystic fibrosis
channelopathies in Cystic fibrosischannelopathies in Cystic fibrosis
channelopathies in Cystic fibrosis
 
Cystic fibrosis dr yusuf imran
Cystic fibrosis dr yusuf imranCystic fibrosis dr yusuf imran
Cystic fibrosis dr yusuf imran
 
Cysticfibrosis
CysticfibrosisCysticfibrosis
Cysticfibrosis
 
Lesson 11 Gene Therapy
Lesson 11   Gene TherapyLesson 11   Gene Therapy
Lesson 11 Gene Therapy
 
Gene therapy teacher
Gene therapy teacherGene therapy teacher
Gene therapy teacher
 
Gene therapy
Gene therapyGene therapy
Gene therapy
 

Similar to Gene Therapy For Cystic Fibrosis

Evidence based management of ipf
Evidence based management of ipfEvidence based management of ipf
Evidence based management of ipfKalai Arasan
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and PresentationsLin Wang
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and PresentationsLin Wang
 
Ton Beniers_publicatielijst overzicht 2015
Ton Beniers_publicatielijst overzicht 2015Ton Beniers_publicatielijst overzicht 2015
Ton Beniers_publicatielijst overzicht 2015Ton Beniers
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and PresentationsLin Wang
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and PresentationsLin Wang
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and PresentationsLin Wang
 
Broomfield Hospital Research Publications During 2017
Broomfield Hospital Research Publications During 2017Broomfield Hospital Research Publications During 2017
Broomfield Hospital Research Publications During 2017Paul Roberts
 
Antimicrobials in Pancreatits.pptx
Antimicrobials in Pancreatits.pptxAntimicrobials in Pancreatits.pptx
Antimicrobials in Pancreatits.pptxDr. Gowtham Krishna
 
Selected Publications
Selected PublicationsSelected Publications
Selected PublicationsShan Yang
 
List of Publications
List of PublicationsList of Publications
List of Publicationsgvanleenders
 
SCT60103 Group 2 Presentation
SCT60103 Group 2 PresentationSCT60103 Group 2 Presentation
SCT60103 Group 2 PresentationShinYiLow
 
Blood transfusion in genomic era
Blood transfusion in genomic eraBlood transfusion in genomic era
Blood transfusion in genomic eraUgochukwu Oduwe
 
CV Volume 2 Publications 02-2016
CV Volume 2 Publications 02-2016CV Volume 2 Publications 02-2016
CV Volume 2 Publications 02-2016Jonathan Finlay
 
10.1164@rccm.201706 1248 le
10.1164@rccm.201706 1248 le10.1164@rccm.201706 1248 le
10.1164@rccm.201706 1248 leJulio A. Diaz M.
 
Pierre_Cauchy_Scientific_CV_complete
Pierre_Cauchy_Scientific_CV_completePierre_Cauchy_Scientific_CV_complete
Pierre_Cauchy_Scientific_CV_completePierre Cauchy
 

Similar to Gene Therapy For Cystic Fibrosis (20)

Evidence based management of ipf
Evidence based management of ipfEvidence based management of ipf
Evidence based management of ipf
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and Presentations
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and Presentations
 
Ton Beniers_publicatielijst overzicht 2015
Ton Beniers_publicatielijst overzicht 2015Ton Beniers_publicatielijst overzicht 2015
Ton Beniers_publicatielijst overzicht 2015
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and Presentations
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and Presentations
 
Publications and Presentations
Publications and PresentationsPublications and Presentations
Publications and Presentations
 
Broomfield Hospital Research Publications During 2017
Broomfield Hospital Research Publications During 2017Broomfield Hospital Research Publications During 2017
Broomfield Hospital Research Publications During 2017
 
CFrecha CV
CFrecha CVCFrecha CV
CFrecha CV
 
Publications and Research Papers
Publications and Research PapersPublications and Research Papers
Publications and Research Papers
 
Antimicrobials in Pancreatits.pptx
Antimicrobials in Pancreatits.pptxAntimicrobials in Pancreatits.pptx
Antimicrobials in Pancreatits.pptx
 
Selected Publications
Selected PublicationsSelected Publications
Selected Publications
 
Publication
PublicationPublication
Publication
 
List of Publications
List of PublicationsList of Publications
List of Publications
 
Annotated Bibliography
Annotated BibliographyAnnotated Bibliography
Annotated Bibliography
 
SCT60103 Group 2 Presentation
SCT60103 Group 2 PresentationSCT60103 Group 2 Presentation
SCT60103 Group 2 Presentation
 
Blood transfusion in genomic era
Blood transfusion in genomic eraBlood transfusion in genomic era
Blood transfusion in genomic era
 
CV Volume 2 Publications 02-2016
CV Volume 2 Publications 02-2016CV Volume 2 Publications 02-2016
CV Volume 2 Publications 02-2016
 
10.1164@rccm.201706 1248 le
10.1164@rccm.201706 1248 le10.1164@rccm.201706 1248 le
10.1164@rccm.201706 1248 le
 
Pierre_Cauchy_Scientific_CV_complete
Pierre_Cauchy_Scientific_CV_completePierre_Cauchy_Scientific_CV_complete
Pierre_Cauchy_Scientific_CV_complete
 

Recently uploaded

Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...
Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...
Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...apidays
 
Artificial Intelligence Chap.5 : Uncertainty
Artificial Intelligence Chap.5 : UncertaintyArtificial Intelligence Chap.5 : Uncertainty
Artificial Intelligence Chap.5 : UncertaintyKhushali Kathiriya
 
Introduction to Multilingual Retrieval Augmented Generation (RAG)
Introduction to Multilingual Retrieval Augmented Generation (RAG)Introduction to Multilingual Retrieval Augmented Generation (RAG)
Introduction to Multilingual Retrieval Augmented Generation (RAG)Zilliz
 
Boost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfBoost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfsudhanshuwaghmare1
 
ICT role in 21st century education and its challenges
ICT role in 21st century education and its challengesICT role in 21st century education and its challenges
ICT role in 21st century education and its challengesrafiqahmad00786416
 
Corporate and higher education May webinar.pptx
Corporate and higher education May webinar.pptxCorporate and higher education May webinar.pptx
Corporate and higher education May webinar.pptxRustici Software
 
Vector Search -An Introduction in Oracle Database 23ai.pptx
Vector Search -An Introduction in Oracle Database 23ai.pptxVector Search -An Introduction in Oracle Database 23ai.pptx
Vector Search -An Introduction in Oracle Database 23ai.pptxRemote DBA Services
 
Cloud Frontiers: A Deep Dive into Serverless Spatial Data and FME
Cloud Frontiers:  A Deep Dive into Serverless Spatial Data and FMECloud Frontiers:  A Deep Dive into Serverless Spatial Data and FME
Cloud Frontiers: A Deep Dive into Serverless Spatial Data and FMESafe Software
 
Exploring Multimodal Embeddings with Milvus
Exploring Multimodal Embeddings with MilvusExploring Multimodal Embeddings with Milvus
Exploring Multimodal Embeddings with MilvusZilliz
 
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemke
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemkeProductAnonymous-April2024-WinProductDiscovery-MelissaKlemke
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemkeProduct Anonymous
 
Architecting Cloud Native Applications
Architecting Cloud Native ApplicationsArchitecting Cloud Native Applications
Architecting Cloud Native ApplicationsWSO2
 
AWS Community Day CPH - Three problems of Terraform
AWS Community Day CPH - Three problems of TerraformAWS Community Day CPH - Three problems of Terraform
AWS Community Day CPH - Three problems of TerraformAndrey Devyatkin
 
Apidays New York 2024 - The value of a flexible API Management solution for O...
Apidays New York 2024 - The value of a flexible API Management solution for O...Apidays New York 2024 - The value of a flexible API Management solution for O...
Apidays New York 2024 - The value of a flexible API Management solution for O...apidays
 
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWER
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWEREMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWER
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWERMadyBayot
 
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...Angeliki Cooney
 
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...apidays
 
WSO2's API Vision: Unifying Control, Empowering Developers
WSO2's API Vision: Unifying Control, Empowering DevelopersWSO2's API Vision: Unifying Control, Empowering Developers
WSO2's API Vision: Unifying Control, Empowering DevelopersWSO2
 
Why Teams call analytics are critical to your entire business
Why Teams call analytics are critical to your entire businessWhy Teams call analytics are critical to your entire business
Why Teams call analytics are critical to your entire businesspanagenda
 

Recently uploaded (20)

+971581248768>> SAFE AND ORIGINAL ABORTION PILLS FOR SALE IN DUBAI AND ABUDHA...
+971581248768>> SAFE AND ORIGINAL ABORTION PILLS FOR SALE IN DUBAI AND ABUDHA...+971581248768>> SAFE AND ORIGINAL ABORTION PILLS FOR SALE IN DUBAI AND ABUDHA...
+971581248768>> SAFE AND ORIGINAL ABORTION PILLS FOR SALE IN DUBAI AND ABUDHA...
 
Understanding the FAA Part 107 License ..
Understanding the FAA Part 107 License ..Understanding the FAA Part 107 License ..
Understanding the FAA Part 107 License ..
 
Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...
Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...
Apidays New York 2024 - Passkeys: Developing APIs to enable passwordless auth...
 
Artificial Intelligence Chap.5 : Uncertainty
Artificial Intelligence Chap.5 : UncertaintyArtificial Intelligence Chap.5 : Uncertainty
Artificial Intelligence Chap.5 : Uncertainty
 
Introduction to Multilingual Retrieval Augmented Generation (RAG)
Introduction to Multilingual Retrieval Augmented Generation (RAG)Introduction to Multilingual Retrieval Augmented Generation (RAG)
Introduction to Multilingual Retrieval Augmented Generation (RAG)
 
Boost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdfBoost Fertility New Invention Ups Success Rates.pdf
Boost Fertility New Invention Ups Success Rates.pdf
 
ICT role in 21st century education and its challenges
ICT role in 21st century education and its challengesICT role in 21st century education and its challenges
ICT role in 21st century education and its challenges
 
Corporate and higher education May webinar.pptx
Corporate and higher education May webinar.pptxCorporate and higher education May webinar.pptx
Corporate and higher education May webinar.pptx
 
Vector Search -An Introduction in Oracle Database 23ai.pptx
Vector Search -An Introduction in Oracle Database 23ai.pptxVector Search -An Introduction in Oracle Database 23ai.pptx
Vector Search -An Introduction in Oracle Database 23ai.pptx
 
Cloud Frontiers: A Deep Dive into Serverless Spatial Data and FME
Cloud Frontiers:  A Deep Dive into Serverless Spatial Data and FMECloud Frontiers:  A Deep Dive into Serverless Spatial Data and FME
Cloud Frontiers: A Deep Dive into Serverless Spatial Data and FME
 
Exploring Multimodal Embeddings with Milvus
Exploring Multimodal Embeddings with MilvusExploring Multimodal Embeddings with Milvus
Exploring Multimodal Embeddings with Milvus
 
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemke
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemkeProductAnonymous-April2024-WinProductDiscovery-MelissaKlemke
ProductAnonymous-April2024-WinProductDiscovery-MelissaKlemke
 
Architecting Cloud Native Applications
Architecting Cloud Native ApplicationsArchitecting Cloud Native Applications
Architecting Cloud Native Applications
 
AWS Community Day CPH - Three problems of Terraform
AWS Community Day CPH - Three problems of TerraformAWS Community Day CPH - Three problems of Terraform
AWS Community Day CPH - Three problems of Terraform
 
Apidays New York 2024 - The value of a flexible API Management solution for O...
Apidays New York 2024 - The value of a flexible API Management solution for O...Apidays New York 2024 - The value of a flexible API Management solution for O...
Apidays New York 2024 - The value of a flexible API Management solution for O...
 
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWER
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWEREMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWER
EMPOWERMENT TECHNOLOGY GRADE 11 QUARTER 2 REVIEWER
 
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...
Biography Of Angeliki Cooney | Senior Vice President Life Sciences | Albany, ...
 
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...
Apidays New York 2024 - APIs in 2030: The Risk of Technological Sleepwalk by ...
 
WSO2's API Vision: Unifying Control, Empowering Developers
WSO2's API Vision: Unifying Control, Empowering DevelopersWSO2's API Vision: Unifying Control, Empowering Developers
WSO2's API Vision: Unifying Control, Empowering Developers
 
Why Teams call analytics are critical to your entire business
Why Teams call analytics are critical to your entire businessWhy Teams call analytics are critical to your entire business
Why Teams call analytics are critical to your entire business
 

Gene Therapy For Cystic Fibrosis

  • 1. Gene Therapy for the Treatment of Cystic Fibrosis Stephanie M. Delgado RISE Program BIOL 3095
  • 2. Cystic Fibrosis Autosomal recessive genetic disorder CFTR gene
  • 3. Gene Therapy What is gene therapy? Objective
  • 4. Degree of correction required Minimal percentage of cells with a non-CF phenotyperequired to normalize the functions of the CF bronchial epithelial layer.
  • 5. Barriers for gene delivery Defense mechanisms Delivery systems
  • 6.
  • 7.
  • 10.
  • 11.
  • 14. Conclusions New design of clinical trials New vectors Gene therapy for CF is still a great hope more than a reality
  • 15. References Buckley SMK , Howe SJ, Sheard V, Ward NJ, Coutelle C, Thrasher AJ, Waddington SN,      McKay TR. 2008. Lentiviral transduction of the murine lung provides efficient pseudotype and developmental stage-dependent cell-specific transgene expression. Gene Therapy 15: 1167–1175 Conese M, Boyd C, Di Gioia S, Auriche C, Ascenzioni F. 2007. Genomic Context Vectors and Artificial Chromosomes for Cystic Fibrosis Gene Therapy. Current Gene Therapy 7: 175-187 Dannhoffer L, Blouquit-Laye S, Regnier A, Chinet T. 2009. Functional Properties of Mixed Cystic Fibrosis and Normal Bronchial Epithelial Cell Cultures. American Journal of Respiratory Cell and Molecular Biology 40: 717–723. Davies JC. 2006. Gene and cell therapy for cystic fibrosis. Paediatric Respiratory Reviews 7(1):S163-S165. Eubanks M. 2001. Gene Therapy for CF. Environmental Health Perspectives 109: A16 Ferrari S,  Griesenbach U, Iida A, Farley R, Wright AM, Zhu J, Munkonge FM, Smith SN, You J, Ban H, Inoue M, Chan M, Singh C, Verdon B, Argent BE, Wainwright B, Jeffery PK, Geddes DM, Porteous DJ, Hyde SC, Gray MA, Hasegawa M, Alton EW. 2007. Sendai virus-mediated CFTR gene transfer to the airway epithelium. Gene Therapy 14: 1371–1379. Griesenbach U, Alton EW. 2007. Progress and Prospects: Gene Therapy Clinical Trials (Part 1). Gene Therapy 14: 1439–1447. Griesenbach U, Eric WF. 2008. Gene transfer to the lung: Lessons learned from more than 2 decades of CF gene therapy. Advanced Drug Delivery Reviews 61: 128–139. Hyde SC, Pringle IA, Abdullah S, Lawton AE, Davies L, Varathalingam A, Nunez-Alonso G, Green A, Bazzani RP, Sumner-Jones SG, Chan M, Li H, Yew NS, Cheng SH,  Boyd C, Davies JC, Griesenbach U, Porteous DJ, Sheppard DN, Munkonge FM, Walton EW, Gill DR. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. 2008. Nature Biotechnology 26: 549-551 Liu X, Luo M, Guo C, Yan Z, Wang Y, Engelhardt JF. 2007. Comparative biology of rAAV transduction in ferret, pig and human airway epithelia. Gene Therapy 14: 1543–1548. O’Sullivan BP, Freedman SD. 2009. Cystic fibrosis. Lancet 373: 1891–904 Ostrowski LE, Yin W, Diggs PS, Rogers TD, O’Neal WK, Grubb BR. 2007. Expression of CFTR from a ciliated cell-specific promoter is ineffective at correcting nasal potential difference in CF mice. Gene Therapy 14: 1492–1501 Price AR, Limberis MP, Wilson JM, Diamond SL. 2007.  Pulmonary delivery of adenovirus vector formulated with dexamethasone–spermine facilitates homologous vector re-administration. Gene Therapy 14: 1594–1604. Rogers CS, Hao Y, Rokhlina T, Samuel M. 2008. Production of CFTR-null and CFTR-[Delta]F508 heterozygous pigs by adeno-associated virus-mediated gene targeting and somatic cell nuclear transfer. Journal of Clinical Investigation 118 (4): 1571-1577 Sumner-Jones SG, Davies LA, Varathalingam A, Gill DR, Hyde SC. 2006. Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways. Gene Therapy 13: 1703–1713. Yu Z, McKay K, Asperen P, Zheng M, Fleming J, Ginn SL, Kizana E, Latham M, Feneley M, Kirkland PD, Rowe P, Lumbers ER, Alexander IE. 2007. Lentivirus vector-mediated gene transfer to the developing bronchiolar airway epithelium in the fetal lamb. The Journal of Gene Medicine 9: 429–439. Ziady AG, Davis PB. 2006. Current prospects for gene therapy of cystic fibrosis. Current Opinion in Pharmacology 6(5):515-521.