Genetic medicines for CF: Hype versus reality
Summary Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair‐based strategies. Pr...
Gespeichert in:
| Veröffentlicht in: | Pediatric pulmonology 2016-10, Vol.51 (S44), p.S5-S17 |
|---|---|
| Hauptverfasser: | , , , , , , , , , , |
| Format: | Artikel |
| Sprache: | eng |
| Schlagworte: | |
| Online-Zugang: | Volltext |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| container_end_page | S17 |
|---|---|
| container_issue | S44 |
| container_start_page | S5 |
| container_title | Pediatric pulmonology |
| container_volume | 51 |
| creator | Alton, Eric W.F.W. Boyd, A. Christopher Davies, Jane C. Gill, Deborah R. Griesenbach, Uta Harrison, Patrick T. Henig, Noreen Higgins, Tracy Hyde, Stephen C. Innes, J. Alastair Korman, Michael S.D. |
| description | Summary
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair‐based strategies. Proof‐of‐concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing‐based strategies are currently at the pre‐clinical phase of development. This review has been written jointly by some of those involved in the various CF “genetic medicine” fields and will summarize the current state‐of‐the‐art, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicine‐based treatment approaches in CF. Pediatr Pulmonol. 2016;51:S5–S17. © 2016 Wiley Periodicals, Inc. |
| doi_str_mv | 10.1002/ppul.23543 |
| format | Article |
| fullrecord | <record><control><sourceid>proquest_cross</sourceid><recordid>TN_cdi_proquest_miscellaneous_1859722833</recordid><sourceformat>XML</sourceformat><sourcesystem>PC</sourcesystem><sourcerecordid>1859722833</sourcerecordid><originalsourceid>FETCH-LOGICAL-c4033-5acb53bc1002c859eccbef3175752c369090be03155af9b72e155da2990b18913</originalsourceid><addsrcrecordid>eNp9kF1LwzAUhoMobk5v_AHSSxE687E0jXdS3CaMOdAheBPa7BSi3VqTVu2_N7PbLr3KIec5D-e8CF0SPCQY09uqaoohZXzEjlCfYClDPJLRMerHgvMwiiPWQ2fOvWPse5Kcoh4VUUQJ5n0UTmADtdHBGlZGmw24IC9tkIzvgmlbQfAF1jUusJAWpm7P0UmeFg4udu8ALccPL8k0nD1NHpP7WahHmLGQpzrjLNPb7XTMJWidQc6I4IJTzSKJJc4AM8J5mstMUPDVKqXSf5NYEjZA1523suVnA65Wa-M0FEW6gbJxinipoDRmzKM3Hapt6ZyFXFXWrFPbKoLVdgG1jUf9xePhq523yfzBB3SfhwdIB3ybAtp_VGqxWM720rCbMa6Gn8NMaj9UJJjg6nU-UdPnBU3mb0TN2S-533zl</addsrcrecordid><sourcetype>Aggregation Database</sourcetype><iscdi>true</iscdi><recordtype>article</recordtype><pqid>1859722833</pqid></control><display><type>article</type><title>Genetic medicines for CF: Hype versus reality</title><source>Wiley Online Library Journals Frontfile Complete</source><creator>Alton, Eric W.F.W. ; Boyd, A. Christopher ; Davies, Jane C. ; Gill, Deborah R. ; Griesenbach, Uta ; Harrison, Patrick T. ; Henig, Noreen ; Higgins, Tracy ; Hyde, Stephen C. ; Innes, J. Alastair ; Korman, Michael S.D.</creator><creatorcontrib>Alton, Eric W.F.W. ; Boyd, A. Christopher ; Davies, Jane C. ; Gill, Deborah R. ; Griesenbach, Uta ; Harrison, Patrick T. ; Henig, Noreen ; Higgins, Tracy ; Hyde, Stephen C. ; Innes, J. Alastair ; Korman, Michael S.D.</creatorcontrib><description>Summary
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair‐based strategies. Proof‐of‐concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing‐based strategies are currently at the pre‐clinical phase of development. This review has been written jointly by some of those involved in the various CF “genetic medicine” fields and will summarize the current state‐of‐the‐art, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicine‐based treatment approaches in CF. Pediatr Pulmonol. 2016;51:S5–S17. © 2016 Wiley Periodicals, Inc.</description><identifier>ISSN: 8755-6863</identifier><identifier>EISSN: 1099-0496</identifier><identifier>DOI: 10.1002/ppul.23543</identifier><identifier>PMID: 27662105</identifier><language>eng</language><publisher>United States: Blackwell Publishing Ltd</publisher><subject>cystic fibrosis (CF) ; gene therapy ; genome editing ; lung</subject><ispartof>Pediatric pulmonology, 2016-10, Vol.51 (S44), p.S5-S17</ispartof><rights>2016 Wiley Periodicals, Inc.</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c4033-5acb53bc1002c859eccbef3175752c369090be03155af9b72e155da2990b18913</citedby><cites>FETCH-LOGICAL-c4033-5acb53bc1002c859eccbef3175752c369090be03155af9b72e155da2990b18913</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2Fppul.23543$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2Fppul.23543$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,780,784,1417,27924,27925,45574,45575</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/27662105$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Alton, Eric W.F.W.</creatorcontrib><creatorcontrib>Boyd, A. Christopher</creatorcontrib><creatorcontrib>Davies, Jane C.</creatorcontrib><creatorcontrib>Gill, Deborah R.</creatorcontrib><creatorcontrib>Griesenbach, Uta</creatorcontrib><creatorcontrib>Harrison, Patrick T.</creatorcontrib><creatorcontrib>Henig, Noreen</creatorcontrib><creatorcontrib>Higgins, Tracy</creatorcontrib><creatorcontrib>Hyde, Stephen C.</creatorcontrib><creatorcontrib>Innes, J. Alastair</creatorcontrib><creatorcontrib>Korman, Michael S.D.</creatorcontrib><title>Genetic medicines for CF: Hype versus reality</title><title>Pediatric pulmonology</title><addtitle>Pediatr Pulmonol</addtitle><description>Summary
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair‐based strategies. Proof‐of‐concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing‐based strategies are currently at the pre‐clinical phase of development. This review has been written jointly by some of those involved in the various CF “genetic medicine” fields and will summarize the current state‐of‐the‐art, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicine‐based treatment approaches in CF. Pediatr Pulmonol. 2016;51:S5–S17. © 2016 Wiley Periodicals, Inc.</description><subject>cystic fibrosis (CF)</subject><subject>gene therapy</subject><subject>genome editing</subject><subject>lung</subject><issn>8755-6863</issn><issn>1099-0496</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><recordid>eNp9kF1LwzAUhoMobk5v_AHSSxE687E0jXdS3CaMOdAheBPa7BSi3VqTVu2_N7PbLr3KIec5D-e8CF0SPCQY09uqaoohZXzEjlCfYClDPJLRMerHgvMwiiPWQ2fOvWPse5Kcoh4VUUQJ5n0UTmADtdHBGlZGmw24IC9tkIzvgmlbQfAF1jUusJAWpm7P0UmeFg4udu8ALccPL8k0nD1NHpP7WahHmLGQpzrjLNPb7XTMJWidQc6I4IJTzSKJJc4AM8J5mstMUPDVKqXSf5NYEjZA1523suVnA65Wa-M0FEW6gbJxinipoDRmzKM3Hapt6ZyFXFXWrFPbKoLVdgG1jUf9xePhq523yfzBB3SfhwdIB3ybAtp_VGqxWM720rCbMa6Gn8NMaj9UJJjg6nU-UdPnBU3mb0TN2S-533zl</recordid><startdate>201610</startdate><enddate>201610</enddate><creator>Alton, Eric W.F.W.</creator><creator>Boyd, A. Christopher</creator><creator>Davies, Jane C.</creator><creator>Gill, Deborah R.</creator><creator>Griesenbach, Uta</creator><creator>Harrison, Patrick T.</creator><creator>Henig, Noreen</creator><creator>Higgins, Tracy</creator><creator>Hyde, Stephen C.</creator><creator>Innes, J. Alastair</creator><creator>Korman, Michael S.D.</creator><general>Blackwell Publishing Ltd</general><scope>BSCLL</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>201610</creationdate><title>Genetic medicines for CF: Hype versus reality</title><author>Alton, Eric W.F.W. ; Boyd, A. Christopher ; Davies, Jane C. ; Gill, Deborah R. ; Griesenbach, Uta ; Harrison, Patrick T. ; Henig, Noreen ; Higgins, Tracy ; Hyde, Stephen C. ; Innes, J. Alastair ; Korman, Michael S.D.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4033-5acb53bc1002c859eccbef3175752c369090be03155af9b72e155da2990b18913</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>cystic fibrosis (CF)</topic><topic>gene therapy</topic><topic>genome editing</topic><topic>lung</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Alton, Eric W.F.W.</creatorcontrib><creatorcontrib>Boyd, A. Christopher</creatorcontrib><creatorcontrib>Davies, Jane C.</creatorcontrib><creatorcontrib>Gill, Deborah R.</creatorcontrib><creatorcontrib>Griesenbach, Uta</creatorcontrib><creatorcontrib>Harrison, Patrick T.</creatorcontrib><creatorcontrib>Henig, Noreen</creatorcontrib><creatorcontrib>Higgins, Tracy</creatorcontrib><creatorcontrib>Hyde, Stephen C.</creatorcontrib><creatorcontrib>Innes, J. Alastair</creatorcontrib><creatorcontrib>Korman, Michael S.D.</creatorcontrib><collection>Istex</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Pediatric pulmonology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Alton, Eric W.F.W.</au><au>Boyd, A. Christopher</au><au>Davies, Jane C.</au><au>Gill, Deborah R.</au><au>Griesenbach, Uta</au><au>Harrison, Patrick T.</au><au>Henig, Noreen</au><au>Higgins, Tracy</au><au>Hyde, Stephen C.</au><au>Innes, J. Alastair</au><au>Korman, Michael S.D.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Genetic medicines for CF: Hype versus reality</atitle><jtitle>Pediatric pulmonology</jtitle><addtitle>Pediatr Pulmonol</addtitle><date>2016-10</date><risdate>2016</risdate><volume>51</volume><issue>S44</issue><spage>S5</spage><epage>S17</epage><pages>S5-S17</pages><issn>8755-6863</issn><eissn>1099-0496</eissn><abstract>Summary
Since identification of the CFTR gene over 25 years ago, gene therapy for cystic fibrosis (CF) has been actively developed. More recently gene therapy has been joined by other forms of “genetic medicines” including mRNA delivery, as well as genome editing and mRNA repair‐based strategies. Proof‐of‐concept that gene therapy can stabilize the progression of CF lung disease has recently been established in a Phase IIb trial. An early phase study to assess the safety and explore efficacy of CFTR mRNA repair is ongoing, while mRNA delivery and genome editing‐based strategies are currently at the pre‐clinical phase of development. This review has been written jointly by some of those involved in the various CF “genetic medicine” fields and will summarize the current state‐of‐the‐art, as well as discuss future developments. Where applicable, it highlights common problems faced by each of the strategies, and also tries to highlight where a specific strategy may have an advantage on the pathway to clinical translation. We hope that this review will contribute to the ongoing discussion about the hype versus reality of genetic medicine‐based treatment approaches in CF. Pediatr Pulmonol. 2016;51:S5–S17. © 2016 Wiley Periodicals, Inc.</abstract><cop>United States</cop><pub>Blackwell Publishing Ltd</pub><pmid>27662105</pmid><doi>10.1002/ppul.23543</doi><tpages>13</tpages><oa>free_for_read</oa></addata></record> |
| fulltext | fulltext |
| identifier | ISSN: 8755-6863 |
| ispartof | Pediatric pulmonology, 2016-10, Vol.51 (S44), p.S5-S17 |
| issn | 8755-6863 1099-0496 |
| language | eng |
| recordid | cdi_proquest_miscellaneous_1859722833 |
| source | Wiley Online Library Journals Frontfile Complete |
| subjects | cystic fibrosis (CF) gene therapy genome editing lung |
| title | Genetic medicines for CF: Hype versus reality |
| url | https://sfx.bib-bvb.de/sfx_tum?ctx_ver=Z39.88-2004&ctx_enc=info:ofi/enc:UTF-8&ctx_tim=2024-12-20T15%3A11%3A31IST&url_ver=Z39.88-2004&url_ctx_fmt=infofi/fmt:kev:mtx:ctx&rfr_id=info:sid/primo.exlibrisgroup.com:primo3-Article-proquest_cross&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.genre=article&rft.atitle=Genetic%20medicines%20for%20CF:%20Hype%20versus%20reality&rft.jtitle=Pediatric%20pulmonology&rft.au=Alton,%20Eric%20W.F.W.&rft.date=2016-10&rft.volume=51&rft.issue=S44&rft.spage=S5&rft.epage=S17&rft.pages=S5-S17&rft.issn=8755-6863&rft.eissn=1099-0496&rft_id=info:doi/10.1002/ppul.23543&rft_dat=%3Cproquest_cross%3E1859722833%3C/proquest_cross%3E%3Curl%3E%3C/url%3E&disable_directlink=true&sfx.directlink=off&sfx.report_link=0&rft_id=info:oai/&rft_pqid=1859722833&rft_id=info:pmid/27662105&rfr_iscdi=true |