A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis
Cystic fibrosis is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. 1 – 4 The normal CFTR gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water. 5 , 6 Mutations in CFTR result in abnorm...
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| Veröffentlicht in: | The New England journal of medicine 1995-09, Vol.333 (13), p.823-831 |
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| container_title | The New England journal of medicine |
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| creator | Knowles, Michael R Hohneker, Kathy W Zhou, Zhaoqing Olsen, John C Noah, Terry L Hu, Ping-Chuan Leigh, Margaret W Engelhardt, John F Edwards, Lloyd J Jones, Kim R Grossman, Mariann Wilson, James M Johnson, Larry G Boucher, Richard C |
| description | Cystic fibrosis is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (
CFTR
) gene.
1
–
4
The normal
CFTR
gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water.
5
,
6
Mutations in
CFTR
result in abnormal secretions that obstruct and ultimately damage epithelium in many areas of the body.
7
The principal cause of death among patients with cystic fibrosis is lung disease. Patients who are homozygous for mutations in the
CFTR
gene have defective cyclic AMP (cAMP)–regulated secretion of chloride
8
,
9
and elevated absorption of sodium . . . |
| doi_str_mv | 10.1056/NEJM199509283331302 |
| format | Article |
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CFTR
) gene.
1
–
4
The normal
CFTR
gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water.
5
,
6
Mutations in
CFTR
result in abnormal secretions that obstruct and ultimately damage epithelium in many areas of the body.
7
The principal cause of death among patients with cystic fibrosis is lung disease. Patients who are homozygous for mutations in the
CFTR
gene have defective cyclic AMP (cAMP)–regulated secretion of chloride
8
,
9
and elevated absorption of sodium . . .</description><identifier>ISSN: 0028-4793</identifier><identifier>EISSN: 1533-4406</identifier><identifier>DOI: 10.1056/NEJM199509283331302</identifier><identifier>PMID: 7544439</identifier><identifier>CODEN: NEJMAG</identifier><language>eng</language><publisher>Boston, MA: Massachusetts Medical Society</publisher><subject>Adenoviruses, Human - genetics ; Adult ; Bacterial infections ; Biological and medical sciences ; Chlorides - metabolism ; Colleges & universities ; Cystic fibrosis ; Cystic Fibrosis - metabolism ; Cystic Fibrosis - therapy ; Cystic Fibrosis Transmembrane Conductance Regulator ; Defective Viruses ; Double-Blind Method ; Epithelium - metabolism ; Epithelium - pathology ; Female ; Gastroenterology. Liver. Pancreas. Abdomen ; Gene therapy ; Gene Transfer Techniques - adverse effects ; Genetic Therapy - adverse effects ; Genetic Therapy - methods ; Genetic Vectors ; Humans ; Ion Transport ; Liver. Biliary tract. Portal circulation. Exocrine pancreas ; Lung diseases ; Male ; Medical sciences ; Membrane Potentials ; Membrane Proteins - genetics ; Mutation ; Nasal Mucosa - metabolism ; Nasal Mucosa - pathology ; Other diseases. Semiology ; RNA, Messenger - analysis</subject><ispartof>The New England journal of medicine, 1995-09, Vol.333 (13), p.823-831</ispartof><rights>Copyright © 1995 Massachusetts Medical Society. All rights reserved.</rights><rights>1995 INIST-CNRS</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c555t-7f2156eb110a80e55da9da3a25f4ddcfbc2317522e27a2e9025019e8669ea17e3</citedby><cites>FETCH-LOGICAL-c555t-7f2156eb110a80e55da9da3a25f4ddcfbc2317522e27a2e9025019e8669ea17e3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://www.nejm.org/doi/pdf/10.1056/NEJM199509283331302$$EPDF$$P50$$Gmms$$H</linktopdf><linktohtml>$$Uhttps://www.proquest.com/docview/223983586?pq-origsite=primo$$EHTML$$P50$$Gproquest$$H</linktohtml><link.rule.ids>314,780,784,2759,2760,26103,27924,27925,52382,54064,64385,64389,72469</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=3676891$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/7544439$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Knowles, Michael R</creatorcontrib><creatorcontrib>Hohneker, Kathy W</creatorcontrib><creatorcontrib>Zhou, Zhaoqing</creatorcontrib><creatorcontrib>Olsen, John C</creatorcontrib><creatorcontrib>Noah, Terry L</creatorcontrib><creatorcontrib>Hu, Ping-Chuan</creatorcontrib><creatorcontrib>Leigh, Margaret W</creatorcontrib><creatorcontrib>Engelhardt, John F</creatorcontrib><creatorcontrib>Edwards, Lloyd J</creatorcontrib><creatorcontrib>Jones, Kim R</creatorcontrib><creatorcontrib>Grossman, Mariann</creatorcontrib><creatorcontrib>Wilson, James M</creatorcontrib><creatorcontrib>Johnson, Larry G</creatorcontrib><creatorcontrib>Boucher, Richard C</creatorcontrib><title>A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis</title><title>The New England journal of medicine</title><addtitle>N Engl J Med</addtitle><description>Cystic fibrosis is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (
CFTR
) gene.
1
–
4
The normal
CFTR
gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water.
5
,
6
Mutations in
CFTR
result in abnormal secretions that obstruct and ultimately damage epithelium in many areas of the body.
7
The principal cause of death among patients with cystic fibrosis is lung disease. Patients who are homozygous for mutations in the
CFTR
gene have defective cyclic AMP (cAMP)–regulated secretion of chloride
8
,
9
and elevated absorption of sodium . . .</description><subject>Adenoviruses, Human - genetics</subject><subject>Adult</subject><subject>Bacterial infections</subject><subject>Biological and medical sciences</subject><subject>Chlorides - metabolism</subject><subject>Colleges & universities</subject><subject>Cystic fibrosis</subject><subject>Cystic Fibrosis - metabolism</subject><subject>Cystic Fibrosis - therapy</subject><subject>Cystic Fibrosis Transmembrane Conductance Regulator</subject><subject>Defective Viruses</subject><subject>Double-Blind Method</subject><subject>Epithelium - metabolism</subject><subject>Epithelium - pathology</subject><subject>Female</subject><subject>Gastroenterology. Liver. Pancreas. Abdomen</subject><subject>Gene therapy</subject><subject>Gene Transfer Techniques - adverse effects</subject><subject>Genetic Therapy - adverse effects</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Vectors</subject><subject>Humans</subject><subject>Ion Transport</subject><subject>Liver. Biliary tract. Portal circulation. Exocrine pancreas</subject><subject>Lung diseases</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Membrane Potentials</subject><subject>Membrane Proteins - genetics</subject><subject>Mutation</subject><subject>Nasal Mucosa - metabolism</subject><subject>Nasal Mucosa - pathology</subject><subject>Other diseases. Semiology</subject><subject>RNA, Messenger - analysis</subject><issn>0028-4793</issn><issn>1533-4406</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1995</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>ABUWG</sourceid><sourceid>AFKRA</sourceid><sourceid>AZQEC</sourceid><sourceid>BEC</sourceid><sourceid>BENPR</sourceid><sourceid>CCPQU</sourceid><sourceid>DWQXO</sourceid><sourceid>GNUQQ</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNp9kNtKAzEQhoMotVafQIQg3slqDpvdzWUpbVXqAazeLulmFlP2UJOs0jvfwTf0SYy0eCXOzTAz3_wz_AgdU3JBiUgu78Y3t1RKQSTLOOeUE7aD-lRwHsUxSXZRnxCWRXEq-T46cG5JQtBY9lAvFXEcc9lH6yEetY23bVWBxo--02vclniooWnfjFVV9AyFb-3Xx-ctaKN8oKbQAJ5b1bgSLDYN9i-A75RTFR6vTCgq09U_Kg_KG2i8w--hi0dr502BJ2ZhW2fcIdorVeXgaJsH6Gkyno-uotn99Ho0nEWFEMJHacmoSGBBKVEZASG0klpxxUQZa12Ui4JxmgrGgKWKgSRMECohSxIJiqbAB-h0o7uy7WsHzufLtrNNOJkzxmXGRZYEiG-gIvzmLJT5yppa2XVOSf5jdv6H2WHrZCvdLWrQvztbd8P8bDtXrlBVGSwrjPvFeJImmaQBO99gde3yBpb1v0e_AfFDlA4</recordid><startdate>19950928</startdate><enddate>19950928</enddate><creator>Knowles, Michael R</creator><creator>Hohneker, Kathy W</creator><creator>Zhou, Zhaoqing</creator><creator>Olsen, John C</creator><creator>Noah, Terry L</creator><creator>Hu, Ping-Chuan</creator><creator>Leigh, Margaret W</creator><creator>Engelhardt, John F</creator><creator>Edwards, Lloyd J</creator><creator>Jones, Kim R</creator><creator>Grossman, Mariann</creator><creator>Wilson, James M</creator><creator>Johnson, Larry G</creator><creator>Boucher, Richard C</creator><general>Massachusetts Medical Society</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>0TZ</scope><scope>7RV</scope><scope>7X7</scope><scope>7XB</scope><scope>8AO</scope><scope>8C1</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AN0</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BEC</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>HCIFZ</scope><scope>K0Y</scope><scope>LK8</scope><scope>M0R</scope><scope>M0T</scope><scope>M1P</scope><scope>M2M</scope><scope>M2O</scope><scope>M2P</scope><scope>M7P</scope><scope>MBDVC</scope><scope>NAPCQ</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>PSYQQ</scope><scope>Q9U</scope></search><sort><creationdate>19950928</creationdate><title>A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis</title><author>Knowles, Michael R ; Hohneker, Kathy W ; Zhou, Zhaoqing ; Olsen, John C ; Noah, Terry L ; Hu, Ping-Chuan ; Leigh, Margaret W ; Engelhardt, John F ; Edwards, Lloyd J ; Jones, Kim R ; Grossman, Mariann ; Wilson, James M ; Johnson, Larry G ; Boucher, Richard C</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c555t-7f2156eb110a80e55da9da3a25f4ddcfbc2317522e27a2e9025019e8669ea17e3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1995</creationdate><topic>Adenoviruses, Human - genetics</topic><topic>Adult</topic><topic>Bacterial infections</topic><topic>Biological and medical sciences</topic><topic>Chlorides - metabolism</topic><topic>Colleges & universities</topic><topic>Cystic fibrosis</topic><topic>Cystic Fibrosis - metabolism</topic><topic>Cystic Fibrosis - therapy</topic><topic>Cystic Fibrosis Transmembrane Conductance Regulator</topic><topic>Defective Viruses</topic><topic>Double-Blind Method</topic><topic>Epithelium - metabolism</topic><topic>Epithelium - pathology</topic><topic>Female</topic><topic>Gastroenterology. Liver. Pancreas. Abdomen</topic><topic>Gene therapy</topic><topic>Gene Transfer Techniques - adverse effects</topic><topic>Genetic Therapy - adverse effects</topic><topic>Genetic Therapy - methods</topic><topic>Genetic Vectors</topic><topic>Humans</topic><topic>Ion Transport</topic><topic>Liver. Biliary tract. Portal circulation. Exocrine pancreas</topic><topic>Lung diseases</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Membrane Potentials</topic><topic>Membrane Proteins - genetics</topic><topic>Mutation</topic><topic>Nasal Mucosa - metabolism</topic><topic>Nasal Mucosa - pathology</topic><topic>Other diseases. Semiology</topic><topic>RNA, Messenger - analysis</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Knowles, Michael R</creatorcontrib><creatorcontrib>Hohneker, Kathy W</creatorcontrib><creatorcontrib>Zhou, Zhaoqing</creatorcontrib><creatorcontrib>Olsen, John C</creatorcontrib><creatorcontrib>Noah, Terry L</creatorcontrib><creatorcontrib>Hu, Ping-Chuan</creatorcontrib><creatorcontrib>Leigh, Margaret W</creatorcontrib><creatorcontrib>Engelhardt, John F</creatorcontrib><creatorcontrib>Edwards, Lloyd J</creatorcontrib><creatorcontrib>Jones, Kim R</creatorcontrib><creatorcontrib>Grossman, Mariann</creatorcontrib><creatorcontrib>Wilson, James M</creatorcontrib><creatorcontrib>Johnson, Larry G</creatorcontrib><creatorcontrib>Boucher, Richard C</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Pharma and Biotech Premium PRO</collection><collection>Nursing & Allied Health Database</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>ProQuest Pharma Collection</collection><collection>Public Health Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>ProQuest Central (Alumni Edition)</collection><collection>ProQuest Central UK/Ireland</collection><collection>British Nursing Database</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>eLibrary</collection><collection>ProQuest Central</collection><collection>Natural Science Collection</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central Korea</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>Research Library Prep</collection><collection>SciTech Premium Collection</collection><collection>New England Journal of Medicine</collection><collection>ProQuest Biological Science Collection</collection><collection>Consumer Health Database</collection><collection>Healthcare Administration Database</collection><collection>Medical Database</collection><collection>Psychology Database</collection><collection>Research Library</collection><collection>Science Database</collection><collection>Biological Science Database</collection><collection>Research Library (Corporate)</collection><collection>Nursing & Allied Health Premium</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>ProQuest Central China</collection><collection>ProQuest One Psychology</collection><collection>ProQuest Central Basic</collection><jtitle>The New England journal of medicine</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Knowles, Michael R</au><au>Hohneker, Kathy W</au><au>Zhou, Zhaoqing</au><au>Olsen, John C</au><au>Noah, Terry L</au><au>Hu, Ping-Chuan</au><au>Leigh, Margaret W</au><au>Engelhardt, John F</au><au>Edwards, Lloyd J</au><au>Jones, Kim R</au><au>Grossman, Mariann</au><au>Wilson, James M</au><au>Johnson, Larry G</au><au>Boucher, Richard C</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis</atitle><jtitle>The New England journal of medicine</jtitle><addtitle>N Engl J Med</addtitle><date>1995-09-28</date><risdate>1995</risdate><volume>333</volume><issue>13</issue><spage>823</spage><epage>831</epage><pages>823-831</pages><issn>0028-4793</issn><eissn>1533-4406</eissn><coden>NEJMAG</coden><abstract>Cystic fibrosis is a recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (
CFTR
) gene.
1
–
4
The normal
CFTR
gene codes for a protein (CFTR) that plays a key part in epithelial transport of salt and water.
5
,
6
Mutations in
CFTR
result in abnormal secretions that obstruct and ultimately damage epithelium in many areas of the body.
7
The principal cause of death among patients with cystic fibrosis is lung disease. Patients who are homozygous for mutations in the
CFTR
gene have defective cyclic AMP (cAMP)–regulated secretion of chloride
8
,
9
and elevated absorption of sodium . . .</abstract><cop>Boston, MA</cop><pub>Massachusetts Medical Society</pub><pmid>7544439</pmid><doi>10.1056/NEJM199509283331302</doi><tpages>9</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 0028-4793 |
| ispartof | The New England journal of medicine, 1995-09, Vol.333 (13), p.823-831 |
| issn | 0028-4793 1533-4406 |
| language | eng |
| recordid | cdi_proquest_journals_223983586 |
| source | MEDLINE; EZB-FREE-00999 freely available EZB journals; ProQuest Central UK/Ireland; New England Journal of Medicine |
| subjects | Adenoviruses, Human - genetics Adult Bacterial infections Biological and medical sciences Chlorides - metabolism Colleges & universities Cystic fibrosis Cystic Fibrosis - metabolism Cystic Fibrosis - therapy Cystic Fibrosis Transmembrane Conductance Regulator Defective Viruses Double-Blind Method Epithelium - metabolism Epithelium - pathology Female Gastroenterology. Liver. Pancreas. Abdomen Gene therapy Gene Transfer Techniques - adverse effects Genetic Therapy - adverse effects Genetic Therapy - methods Genetic Vectors Humans Ion Transport Liver. Biliary tract. Portal circulation. Exocrine pancreas Lung diseases Male Medical sciences Membrane Potentials Membrane Proteins - genetics Mutation Nasal Mucosa - metabolism Nasal Mucosa - pathology Other diseases. Semiology RNA, Messenger - analysis |
| title | A Controlled Study of Adenoviral-Vector–Mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis |
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