Stem cell gene therapy for Fanconi anemia: report from the 1st International Fanconi anemia gene therapy working group meeting
Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could po...
Saved in:
| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article (Journal) Conference Paper |
| Language: | English |
| Published: |
2011
|
| In: |
Molecular therapy
Year: 2011, Volume: 19, Issue: 7, Pages: 1193-1198 |
| ISSN: | 1525-0024 |
| DOI: | 10.1038/mt.2011.78 |
| Online Access: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1038/mt.2011.78 Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S1525001616324856 
|
| Author Notes: | Jakub Tolar, Jennifer E Adair, Michael Antoniou, Cynthia C Bartholomae, Pamela S Becker, Bruce R Blazar, Juan Bueren, Thomas Carroll, Marina Cavazzana-Calvo, D Wade Clapp, Robert Dalgleish, Anne Galy, H Bobby Gaspar, Helmut Hanenberg, Christof Von Kalle, Hans-Peter Kiem, Dirk Lindeman, Luigi Naldini, Susana Navarro, Raffaele Renella, Paula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E Wagner, David A Williams and Adrian J Thrasher |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1821591372 | ||
| 003 | DE-627 | ||
| 005 | 20230710102857.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 221110s2011 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1038/mt.2011.78 |2 doi | |
| 035 | |a (DE-627)1821591372 | ||
| 035 | |a (DE-599)KXP1821591372 | ||
| 035 | |a (OCoLC)1389753227 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 33 |2 sdnb | ||
| 100 | 1 | |a Tolar, Jakub |e VerfasserIn |0 (DE-588)1272469069 |0 (DE-627)1821594827 |4 aut | |
| 245 | 1 | 0 | |a Stem cell gene therapy for Fanconi anemia |b report from the 1st International Fanconi anemia gene therapy working group meeting |c Jakub Tolar, Jennifer E Adair, Michael Antoniou, Cynthia C Bartholomae, Pamela S Becker, Bruce R Blazar, Juan Bueren, Thomas Carroll, Marina Cavazzana-Calvo, D Wade Clapp, Robert Dalgleish, Anne Galy, H Bobby Gaspar, Helmut Hanenberg, Christof Von Kalle, Hans-Peter Kiem, Dirk Lindeman, Luigi Naldini, Susana Navarro, Raffaele Renella, Paula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E Wagner, David A Williams and Adrian J Thrasher |
| 264 | 1 | |c 2011 | |
| 300 | |a 6 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Gesehen am 10.11.2022 | ||
| 500 | |a Erstmals am 14. Dezember 2016 online veröffentlicht | ||
| 520 | |a Survival rates after allogeneic hematopoietic cell transplantation (HCT) for Fanconi anemia (FA) have increased dramatically since 2000. However, the use of autologous stem cell gene therapy, whereby the patient's own blood stem cells are modified to express the wild-type gene product, could potentially avoid the early and late complications of allogeneic HCT. Over the last decades, gene therapy has experienced a high degree of optimism interrupted by periods of diminished expectation. Optimism stems from recent examples of successful gene correction in several congenital immunodeficiencies, whereas diminished expectations come from the realization that gene therapy will not be free of side effects. The goal of the 1st International Fanconi Anemia Gene Therapy Working Group Meeting was to determine the optimal strategy for moving stem cell gene therapy into clinical trials for individuals with FA. To this end, key investigators examined vector design, transduction method, criteria for large-scale clinical-grade vector manufacture, hematopoietic cell preparation, and eligibility criteria for FA patients most likely to benefit. The report summarizes the roadmap for the development of gene therapy for FA. | ||
| 700 | 1 | |a Adair, Jennifer E |e VerfasserIn |4 aut | |
| 700 | 1 | |a Antoniou, Michael |e VerfasserIn |4 aut | |
| 700 | 1 | |a Bartholomä, Cynthia C. |e VerfasserIn |0 (DE-588)1036481042 |0 (DE-627)751079243 |0 (DE-576)384489524 |4 aut | |
| 700 | 1 | |a Becker, Pamela S |e VerfasserIn |4 aut | |
| 700 | 1 | |a Blazar, Bruce R |e VerfasserIn |4 aut | |
| 700 | 1 | |a Bueren, Juan |e VerfasserIn |4 aut | |
| 700 | 1 | |a Carroll, Thomas |e VerfasserIn |4 aut | |
| 700 | 1 | |a Cavazzana-Calvo, Marina |e VerfasserIn |4 aut | |
| 700 | 1 | |a Clapp, D Wade |e VerfasserIn |4 aut | |
| 700 | 1 | |a Dalgleish, Robert |e VerfasserIn |4 aut | |
| 700 | 1 | |a Galy, Anne |e VerfasserIn |4 aut | |
| 700 | 1 | |a Gaspar, H Bobby |e VerfasserIn |4 aut | |
| 700 | 1 | |a Hanenberg, Helmut |e VerfasserIn |4 aut | |
| 700 | 1 | |a Kalle, Christof von |d 1962- |e VerfasserIn |0 (DE-588)1036481115 |0 (DE-627)75107926X |0 (DE-576)168957396 |4 aut | |
| 700 | 1 | |a Kiem, Hans-Peter |e VerfasserIn |4 aut | |
| 700 | 1 | |a Lindeman, Dirk |e VerfasserIn |4 aut | |
| 700 | 1 | |a Naldini, Luigi |e VerfasserIn |4 aut | |
| 700 | 1 | |a Navarro, Susana |e VerfasserIn |4 aut | |
| 700 | 1 | |a Renella, Raffaele |e VerfasserIn |4 aut | |
| 700 | 1 | |a Rio, Paula |e VerfasserIn |4 aut | |
| 700 | 1 | |a Sevilla, Julián |e VerfasserIn |4 aut | |
| 700 | 1 | |a Schmidt, Manfred |d 1967-2022 |e VerfasserIn |0 (DE-588)122922301 |0 (DE-627)706092430 |0 (DE-576)293477620 |4 aut | |
| 700 | 1 | |a Verhoeyen, Els |e VerfasserIn |4 aut | |
| 700 | 1 | |a Wagner, John E |e VerfasserIn |4 aut | |
| 700 | 1 | |a Williams, David A |e VerfasserIn |4 aut | |
| 700 | 1 | |a Thrasher, Adrian J |e VerfasserIn |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Molecular therapy |d Amsterdam : Elsevier, 2000 |g 19(2011), 7 vom: Juli, Seite 1193-1198 |h Online-Ressource |w (DE-627)320415198 |w (DE-600)2001818-6 |w (DE-576)104344695 |x 1525-0024 |7 nnas |a Stem cell gene therapy for Fanconi anemia report from the 1st International Fanconi anemia gene therapy working group meeting |
| 773 | 1 | 8 | |g volume:19 |g year:2011 |g number:7 |g month:07 |g pages:1193-1198 |g extent:6 |a Stem cell gene therapy for Fanconi anemia report from the 1st International Fanconi anemia gene therapy working group meeting |
| 856 | 4 | 0 | |u https://doi.org/10.1038/mt.2011.78 |x Verlag |x Resolving-System |z lizenzpflichtig |3 Volltext |
| 856 | 4 | 0 | |u https://www.sciencedirect.com/science/article/pii/S1525001616324856 |x Verlag |z lizenzpflichtig |3 Volltext |
| 951 | |a AR | ||
| 992 | |a 20221110 | ||
| 993 | |a ConferencePaper | ||
| 994 | |a 2011 | ||
| 998 | |g 122922301 |a Schmidt, Manfred |m 122922301:Schmidt, Manfred |d 140000 |e 140000PS122922301 |k 0/140000/ |p 23 | ||
| 998 | |g 1036481115 |a Kalle, Christof von |m 1036481115:Kalle, Christof von |d 910000 |e 910000PK1036481115 |k 0/910000/ |p 15 | ||
| 998 | |g 1036481042 |a Bartholomä, Cynthia C. |m 1036481042:Bartholomä, Cynthia C. |d 50000 |e 50000PB1036481042 |k 0/50000/ |p 4 | ||
| 999 | |a KXP-PPN1821591372 |e 4208525770 | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"relHost":[{"id":{"issn":["1525-0024"],"zdb":["2001818-6"],"eki":["320415198"]},"origin":[{"publisherPlace":"Amsterdam ; New York, NY ; Orlando, Fla.","publisher":"Elsevier ; Nature Publ. Group ; Acad. Press","dateIssuedKey":"2000","dateIssuedDisp":"2000-"}],"part":{"pages":"1193-1198","volume":"19","extent":"6","text":"19(2011), 7 vom: Juli, Seite 1193-1198","issue":"7","year":"2011"},"recId":"320415198","disp":"Stem cell gene therapy for Fanconi anemia report from the 1st International Fanconi anemia gene therapy working group meetingMolecular therapy","title":[{"title":"Molecular therapy","subtitle":"official journal of the American Society of Gene Therapy","title_sort":"Molecular therapy"}],"type":{"bibl":"periodical","media":"Online-Ressource"},"corporate":[{"display":"American Society of Gene Therapy","role":"isb"}],"note":["Gesehen am 28.05.2020"],"pubHistory":["1.2000 -"],"language":["eng"],"physDesc":[{"extent":"Online-Ressource"}]}],"note":["Gesehen am 10.11.2022","Erstmals am 14. Dezember 2016 online veröffentlicht"],"id":{"doi":["10.1038/mt.2011.78"],"eki":["1821591372"]},"origin":[{"dateIssuedDisp":"2011","dateIssuedKey":"2011"}],"name":{"displayForm":["Jakub Tolar, Jennifer E Adair, Michael Antoniou, Cynthia C Bartholomae, Pamela S Becker, Bruce R Blazar, Juan Bueren, Thomas Carroll, Marina Cavazzana-Calvo, D Wade Clapp, Robert Dalgleish, Anne Galy, H Bobby Gaspar, Helmut Hanenberg, Christof Von Kalle, Hans-Peter Kiem, Dirk Lindeman, Luigi Naldini, Susana Navarro, Raffaele Renella, Paula Rio, Julián Sevilla, Manfred Schmidt, Els Verhoeyen, John E Wagner, David A Williams and Adrian J Thrasher"]},"recId":"1821591372","person":[{"display":"Tolar, Jakub","role":"aut","given":"Jakub","family":"Tolar"},{"given":"Jennifer E","role":"aut","family":"Adair","display":"Adair, Jennifer E"},{"given":"Michael","role":"aut","family":"Antoniou","display":"Antoniou, Michael"},{"display":"Bartholomä, Cynthia C.","role":"aut","given":"Cynthia C.","family":"Bartholomä"},{"display":"Becker, Pamela S","family":"Becker","given":"Pamela S","role":"aut"},{"family":"Blazar","given":"Bruce R","role":"aut","display":"Blazar, Bruce R"},{"family":"Bueren","given":"Juan","role":"aut","display":"Bueren, Juan"},{"family":"Carroll","given":"Thomas","role":"aut","display":"Carroll, Thomas"},{"display":"Cavazzana-Calvo, Marina","given":"Marina","role":"aut","family":"Cavazzana-Calvo"},{"display":"Clapp, D Wade","family":"Clapp","role":"aut","given":"D Wade"},{"display":"Dalgleish, Robert","family":"Dalgleish","given":"Robert","role":"aut"},{"display":"Galy, Anne","given":"Anne","role":"aut","family":"Galy"},{"display":"Gaspar, H Bobby","family":"Gaspar","given":"H Bobby","role":"aut"},{"display":"Hanenberg, Helmut","family":"Hanenberg","role":"aut","given":"Helmut"},{"role":"aut","given":"Christof von","family":"Kalle","display":"Kalle, Christof von"},{"family":"Kiem","given":"Hans-Peter","role":"aut","display":"Kiem, Hans-Peter"},{"display":"Lindeman, Dirk","family":"Lindeman","given":"Dirk","role":"aut"},{"display":"Naldini, Luigi","family":"Naldini","given":"Luigi","role":"aut"},{"display":"Navarro, Susana","family":"Navarro","role":"aut","given":"Susana"},{"given":"Raffaele","role":"aut","family":"Renella","display":"Renella, Raffaele"},{"role":"aut","given":"Paula","family":"Rio","display":"Rio, Paula"},{"given":"Julián","role":"aut","family":"Sevilla","display":"Sevilla, Julián"},{"display":"Schmidt, Manfred","family":"Schmidt","role":"aut","given":"Manfred"},{"role":"aut","given":"Els","family":"Verhoeyen","display":"Verhoeyen, Els"},{"family":"Wagner","role":"aut","given":"John E","display":"Wagner, John E"},{"display":"Williams, David A","role":"aut","given":"David A","family":"Williams"},{"given":"Adrian J","role":"aut","family":"Thrasher","display":"Thrasher, Adrian J"}],"title":[{"title_sort":"Stem cell gene therapy for Fanconi anemia","title":"Stem cell gene therapy for Fanconi anemia","subtitle":"report from the 1st International Fanconi anemia gene therapy working group meeting"}],"physDesc":[{"extent":"6 S."}],"language":["eng"],"type":{"media":"Online-Ressource","bibl":"article-journal"}} | ||
| SRT | |a TOLARJAKUBSTEMCELLGE2011 | ||