Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants
Gene delivery vectors derived from Adeno-associated virus (AAV) are one of the most promising tools for the treatment of genetic diseases, evidenced by encouraging clinical data and the approval of several AAV gene therapies. Two major reasons for the success of AAV vectors are (i) the prior isolati...
Gespeichert in:
| Hauptverfasser: | , , , , , , , , , , |
|---|---|
| Dokumenttyp: | Article (Journal) Video |
| Sprache: | Englisch |
| Veröffentlicht: |
October 18th, 2022
|
| In: |
JoVE. Video journal
Year: 2022, Heft: 188, Pages: 1-29 |
| ISSN: | 1940-087X |
| DOI: | 10.3791/64389 |
| Schlagworte: | |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.3791/64389 Verlag, lizenzpflichtig, Volltext: https://www.jove.com/t/64389/isolation-of-next--generation-gene-therapy-vectors-through-engineering-barcoding-and-screening-of-adeno--associated-virus-aav-capsid-variants 
|
| Verfasserangaben: | Kleopatra Rapti, Olena Maiakovska, Jonas Becker, Joanna Szumska, Margarita Zayas, Felix Bubeck, Jixin Liu, Emma Gerstmann, Chiara Krämer, Ellen Wiedtke, Dirk Grimm |
MARC
| LEADER | 00000cga a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1837285659 | ||
| 003 | DE-627 | ||
| 005 | 20230706232129.0 | ||
| 006 | m o | | | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 230222s2022 xx ||| | o ||eng c | ||
| 024 | 7 | |a 10.3791/64389 |2 doi | |
| 035 | |a (DE-627)1837285659 | ||
| 035 | |a (DE-599)KXP1837285659 | ||
| 035 | |a (OCoLC)1389535482 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 32 |2 sdnb | ||
| 100 | 1 | |a Rapti, Kleopatra |e VerfasserIn |0 (DE-588)1160692459 |0 (DE-627)1024099180 |0 (DE-576)506128512 |4 aut | |
| 245 | 1 | 0 | |a Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants |c Kleopatra Rapti, Olena Maiakovska, Jonas Becker, Joanna Szumska, Margarita Zayas, Felix Bubeck, Jixin Liu, Emma Gerstmann, Chiara Krämer, Ellen Wiedtke, Dirk Grimm |
| 264 | 1 | |c October 18th, 2022 | |
| 300 | |a 1 Online-Ressource (1 Videodatei, 9:20 min) |b farbig | ||
| 300 | |a 29 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 336 | |a zweidimensionales bewegtes Bild |b tdi |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Enthält auch Textversion | ||
| 500 | |a Wissenschaftlicher Film. Deutschland. 2022 | ||
| 520 | |a Gene delivery vectors derived from Adeno-associated virus (AAV) are one of the most promising tools for the treatment of genetic diseases, evidenced by encouraging clinical data and the approval of several AAV gene therapies. Two major reasons for the success of AAV vectors are (i) the prior isolation of various naturally occurring viral serotypes with distinct properties, and (ii) the subsequent establishment of powerful technologies for their molecular engineering and repurposing in high throughput. Further boosting the potential of these techniques are recently implemented strategies for barcoding selected AAV capsids on the DNA and RNA level, permitting their comprehensive and parallel in vivo stratification in all major organs and cell types in a single animal. Here, we present a basic pipeline encompassing this set of complementary avenues, using AAV peptide display to represent the diverse arsenal of available capsid engineering technologies. Accordingly, we first describe the pivotal steps for the generation of an AAV peptide display library for the in vivo selection of candidates with desired properties, followed by a demonstration of how to barcode the most interesting capsid variants for secondary in vivo screening. Next, we exemplify the methodology for the creation of libraries for next-generation sequencing (NGS), including barcode amplification and adaptor ligation, before concluding with an overview of the most critical steps during NGS data analysis. As the protocols reported here are versatile and adaptable, researchers can easily harness them to enrich the optimal AAV capsid variants in their favorite disease model and for gene therapy applications. | ||
| 655 | 7 | |a Film |0 (DE-588)4017102-4 |0 (DE-627)104559683 |0 (DE-576)208918531 |2 gnd-content | |
| 700 | 1 | |a Maiakovska, Olena |d 1993- |e VerfasserIn |0 (DE-588)1249736234 |0 (DE-627)1786289199 |4 aut | |
| 700 | 1 | |a Becker, Jonas |d 1995- |e VerfasserIn |0 (DE-588)1262427800 |0 (DE-627)1810114853 |4 aut | |
| 700 | 1 | |a Szumska, Joanna |e VerfasserIn |0 (DE-588)1200511336 |0 (DE-627)1683650352 |4 aut | |
| 700 | 1 | |a Zayas López, Margarita Laura |e VerfasserIn |0 (DE-588)1017966826 |0 (DE-627)679810862 |0 (DE-576)353867586 |4 aut | |
| 700 | 1 | |a Bubeck, Felix |d 1992- |e VerfasserIn |0 (DE-588)1172685525 |0 (DE-627)104131955X |0 (DE-576)514732695 |4 aut | |
| 700 | 1 | |a Liu, Jixin |e VerfasserIn |0 (DE-588)1281645044 |0 (DE-627)1837292086 |4 aut | |
| 700 | 1 | |a Gerstmann, Emma |e VerfasserIn |0 (DE-588)1281640344 |0 (DE-627)1837289093 |4 aut | |
| 700 | 1 | |a Krämer, Chiara |e VerfasserIn |0 (DE-588)1189223422 |0 (DE-627)166796206X |4 aut | |
| 700 | 1 | |a Wiedtke, Ellen |e VerfasserIn |0 (DE-588)1071738372 |0 (DE-627)826301347 |0 (DE-576)433285206 |4 aut | |
| 700 | 1 | |a Grimm, Dirk |e VerfasserIn |0 (DE-588)1016476671 |0 (DE-627)70553376X |0 (DE-576)352260645 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t JoVE. Video journal |d [Erscheinungsort nicht ermittelbar] : [Verlag nicht ermittelbar], 2006 |g (2022), 188, Artikel-ID e64389, Seite 1-29 |h Online-Ressource |w (DE-627)521454778 |w (DE-600)2259946-0 |w (DE-576)338522980 |x 1940-087X |7 nngs |a Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants |
| 773 | 1 | 8 | |g year:2022 |g number:188 |g elocationid:e64389 |g pages:1-29 |g extent:29 |a Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants |
| 856 | 4 | 0 | |u https://doi.org/10.3791/64389 |x Verlag |z lizenzpflichtig |3 Volltext |
| 856 | 4 | 0 | |u https://www.jove.com/t/64389/isolation-of-next--generation-gene-therapy-vectors-through-engineering-barcoding-and-screening-of-adeno--associated-virus-aav-capsid-variants |x Verlag |z lizenzpflichtig |3 Volltext |
| 935 | |c vide | ||
| 937 | |a Wissenschaftlicher Film |b Deutschland |c 2022 | ||
| 951 | |a AR | ||
| 992 | |a 20230222 | ||
| 993 | |a Video | ||
| 994 | |a 2022 | ||
| 998 | |g 1016476671 |a Grimm, Dirk |m 1016476671:Grimm, Dirk |d 910000 |d 911700 |e 910000PG1016476671 |e 911700PG1016476671 |k 0/910000/ |k 1/910000/911700/ |p 11 |y j | ||
| 998 | |g 1071738372 |a Wiedtke, Ellen |m 1071738372:Wiedtke, Ellen |d 700000 |d 716000 |e 700000PW1071738372 |e 716000PW1071738372 |k 0/700000/ |k 1/700000/716000/ |p 10 | ||
| 998 | |g 1189223422 |a Krämer, Chiara |m 1189223422:Krämer, Chiara |d 700000 |d 716000 |e 700000PK1189223422 |e 716000PK1189223422 |k 0/700000/ |k 1/700000/716000/ |p 9 | ||
| 998 | |g 1281640344 |a Gerstmann, Emma |m 1281640344:Gerstmann, Emma |d 700000 |d 716000 |e 700000PG1281640344 |e 716000PG1281640344 |k 0/700000/ |k 1/700000/716000/ |p 8 | ||
| 998 | |g 1281645044 |a Liu, Jixin |m 1281645044:Liu, Jixin |d 910000 |d 911700 |e 910000PL1281645044 |e 911700PL1281645044 |k 0/910000/ |k 1/910000/911700/ |p 7 | ||
| 998 | |g 1172685525 |a Bubeck, Felix |m 1172685525:Bubeck, Felix |d 910000 |d 911700 |e 910000PB1172685525 |e 911700PB1172685525 |k 0/910000/ |k 1/910000/911700/ |p 6 | ||
| 998 | |g 1017966826 |a Zayas López, Margarita Laura |m 1017966826:Zayas López, Margarita Laura |d 910000 |d 911700 |e 910000PZ1017966826 |e 911700PZ1017966826 |k 0/910000/ |k 1/910000/911700/ |p 5 | ||
| 998 | |g 1200511336 |a Szumska, Joanna |m 1200511336:Szumska, Joanna |p 4 | ||
| 998 | |g 1262427800 |a Becker, Jonas |m 1262427800:Becker, Jonas |d 910000 |d 911700 |e 910000PB1262427800 |e 911700PB1262427800 |k 0/910000/ |k 1/910000/911700/ |p 3 | ||
| 998 | |g 1249736234 |a Maiakovska, Olena |m 1249736234:Maiakovska, Olena |d 910000 |d 911700 |e 910000PM1249736234 |e 911700PM1249736234 |k 0/910000/ |k 1/910000/911700/ |p 2 | ||
| 998 | |g 1160692459 |a Rapti, Kleopatra |m 1160692459:Rapti, Kleopatra |d 910000 |d 911700 |e 910000PR1160692459 |e 911700PR1160692459 |k 0/910000/ |k 1/910000/911700/ |p 1 |x j | ||
| 999 | |a KXP-PPN1837285659 |e 4275514173 | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"origin":[{"dateIssuedDisp":"October 18th, 2022","dateIssuedKey":"2022"}],"note":["Enthält auch Textversion","Wissenschaftlicher Film. Deutschland. 2022"],"name":{"displayForm":["Kleopatra Rapti, Olena Maiakovska, Jonas Becker, Joanna Szumska, Margarita Zayas, Felix Bubeck, Jixin Liu, Emma Gerstmann, Chiara Krämer, Ellen Wiedtke, Dirk Grimm"]},"recId":"1837285659","title":[{"title":"Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants","title_sort":"Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variants"}],"relHost":[{"pubHistory":["1.2006 -"],"part":{"year":"2022","extent":"29","issue":"188","pages":"1-29","text":"(2022), 188, Artikel-ID e64389, Seite 1-29"},"type":{"media":"Online-Ressource","bibl":"periodical"},"id":{"eki":["521454778"],"zdb":["2259946-0"],"issn":["1940-087X"]},"origin":[{"publisherPlace":"[Erscheinungsort nicht ermittelbar]","dateIssuedKey":"2006","dateIssuedDisp":"2006-","publisher":"[Verlag nicht ermittelbar]"}],"recId":"521454778","titleAlt":[{"title":"JoVE journal"},{"title":"JoVE journal Biology"},{"title":"JoVE journal Medicine"},{"title":"JoVE journal Behavior"},{"title":"JoVE journal Biochemistry"},{"title":"JoVE journal Bioengineering"},{"title":"JoVE journal Cancer research"},{"title":"JoVE journal Chemistry"},{"title":"JoVE journal Developmental biology"},{"title":"JoVE journal Engineering"},{"title":"JoVE journal Environment"},{"title":"JoVE journal Genetics"},{"title":"JoVE journal Immunology and infection"},{"title":"JoVE journal Neuroscience"},{"title":"Journal of visualized experiments"}],"title":[{"title_sort":"JoVE","partname":"Video journal","title":"JoVE"}],"physDesc":[{"extent":"Online-Ressource"}],"language":["eng"],"note":["Gesehen am 10.02.25","Inhaltliche Gliederung in: Behavior; Biochemistry; Bioengineering; Biology; Cancer research; Chemistry; Developmental biology; Engineering; Environment; Genetics; Immunology and infection; Medicine; Neuroscience"],"disp":"Isolation of next-generation gene therapy vectors through engineering, barcoding, and screening of Adeno-Associated Virus (AAV) capsid variantsJoVE. Video journal"}],"physDesc":[{"extent":"29 S.","noteIll":"farbig"}],"id":{"doi":["10.3791/64389"],"eki":["1837285659"]},"type":{"bibl":"article-journal","media":"Online-Ressource"},"person":[{"given":"Kleopatra","family":"Rapti","role":"aut","display":"Rapti, Kleopatra"},{"role":"aut","display":"Maiakovska, Olena","given":"Olena","family":"Maiakovska"},{"given":"Jonas","family":"Becker","role":"aut","display":"Becker, Jonas"},{"display":"Szumska, Joanna","role":"aut","family":"Szumska","given":"Joanna"},{"given":"Margarita Laura","family":"Zayas López","role":"aut","display":"Zayas López, Margarita Laura"},{"family":"Bubeck","given":"Felix","display":"Bubeck, Felix","role":"aut"},{"display":"Liu, Jixin","role":"aut","family":"Liu","given":"Jixin"},{"role":"aut","display":"Gerstmann, Emma","given":"Emma","family":"Gerstmann"},{"given":"Chiara","family":"Krämer","role":"aut","display":"Krämer, Chiara"},{"given":"Ellen","family":"Wiedtke","role":"aut","display":"Wiedtke, Ellen"},{"role":"aut","display":"Grimm, Dirk","given":"Dirk","family":"Grimm"}],"language":["eng"]} | ||
| SRT | |a RAPTIKLEOPISOLATIONO1820 | ||