mRNA-Based Genetic Reprogramming
The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized regenerative treatments based on immunologically compatible material derived from a patient's own cells might be realized in the not-to...
Gespeichert in:
| Veröffentlicht in: | Molecular therapy Jg. 27; H. 4; S. 729 |
|---|---|
| Hauptverfasser: | , |
| Format: | Journal Article |
| Sprache: | Englisch |
| Veröffentlicht: |
United States
10.04.2019
|
| Schlagworte: | |
| ISSN: | 1525-0024, 1525-0024 |
| Online-Zugang: | Weitere Angaben |
| Tags: |
Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!
|
| Abstract | The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized regenerative treatments based on immunologically compatible material derived from a patient's own cells might be realized in the not-too-distant future. A major barrier to the clinical use of induced pluripotent stem cells (iPSCs) was initially presented by the need to employ integrating viral vectors to express the factors that induce an embryonic gene expression profile, which entails potentially oncogenic alteration of the normal genome. Several "non-integrating" reprogramming systems have been developed over the last decade to address this problem. Among these techniques, mRNA reprogramming is the most unambiguously "footprint-free," most productive, and perhaps the best suited to clinical production of stem cells. Herein, we discuss the origins of the mRNA-based reprogramming system, its benefits and drawbacks, recent technical improvements that simplify its application, and the status of current efforts to industrialize this approach to mass-produce human stem cells for the clinic. |
|---|---|
| AbstractList | The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized regenerative treatments based on immunologically compatible material derived from a patient's own cells might be realized in the not-too-distant future. A major barrier to the clinical use of induced pluripotent stem cells (iPSCs) was initially presented by the need to employ integrating viral vectors to express the factors that induce an embryonic gene expression profile, which entails potentially oncogenic alteration of the normal genome. Several "non-integrating" reprogramming systems have been developed over the last decade to address this problem. Among these techniques, mRNA reprogramming is the most unambiguously "footprint-free," most productive, and perhaps the best suited to clinical production of stem cells. Herein, we discuss the origins of the mRNA-based reprogramming system, its benefits and drawbacks, recent technical improvements that simplify its application, and the status of current efforts to industrialize this approach to mass-produce human stem cells for the clinic. The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized regenerative treatments based on immunologically compatible material derived from a patient's own cells might be realized in the not-too-distant future. A major barrier to the clinical use of induced pluripotent stem cells (iPSCs) was initially presented by the need to employ integrating viral vectors to express the factors that induce an embryonic gene expression profile, which entails potentially oncogenic alteration of the normal genome. Several "non-integrating" reprogramming systems have been developed over the last decade to address this problem. Among these techniques, mRNA reprogramming is the most unambiguously "footprint-free," most productive, and perhaps the best suited to clinical production of stem cells. Herein, we discuss the origins of the mRNA-based reprogramming system, its benefits and drawbacks, recent technical improvements that simplify its application, and the status of current efforts to industrialize this approach to mass-produce human stem cells for the clinic.The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized regenerative treatments based on immunologically compatible material derived from a patient's own cells might be realized in the not-too-distant future. A major barrier to the clinical use of induced pluripotent stem cells (iPSCs) was initially presented by the need to employ integrating viral vectors to express the factors that induce an embryonic gene expression profile, which entails potentially oncogenic alteration of the normal genome. Several "non-integrating" reprogramming systems have been developed over the last decade to address this problem. Among these techniques, mRNA reprogramming is the most unambiguously "footprint-free," most productive, and perhaps the best suited to clinical production of stem cells. Herein, we discuss the origins of the mRNA-based reprogramming system, its benefits and drawbacks, recent technical improvements that simplify its application, and the status of current efforts to industrialize this approach to mass-produce human stem cells for the clinic. |
| Author | Warren, Luigi Lin, Cory |
| Author_xml | – sequence: 1 givenname: Luigi surname: Warren fullname: Warren, Luigi email: luigi.warren@cellular-reprograming.com organization: Cellular Reprogramming, Inc., Pasadena, CA, USA. Electronic address: luigi.warren@cellular-reprograming.com – sequence: 2 givenname: Cory surname: Lin fullname: Lin, Cory organization: Cellular Reprogramming, Inc., Pasadena, CA, USA |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/30598301$$D View this record in MEDLINE/PubMed |
| BookMark | eNpNj01Lw0AYhBep2A_9BYLk6CXxffcjyR5rqVUoCkXPYbN5t6Zkk5pND_33VqzgaWbgYYaZslHbtcTYLUKCgOnDLjn64ZMSDpgnyBMAfcEmqLiKAbgc_fNjNg1hB4CodHrFxgKUzgXghEV-8zqPH02gKlpRS0Ntow3t-27bG-_rdnvNLp1pAt2cdcY-npbvi-d4_bZ6WczXsZV5NsTWKmm1c5mWJXKAjHNTghFoBFitBKGDn6wcOpsqLiqEqgSXlxLSUks-Y_e_vaftrwOFofB1sNQ0pqXuEAqOKZdaoMhO6N0ZPZSeqmLf1970x-LvFf8GLF1PZA |
| CitedBy_id | crossref_primary_10_1155_2020_3692937 crossref_primary_10_1016_j_omtn_2022_10_003 crossref_primary_10_1007_s12015_024_10783_7 crossref_primary_10_1016_j_addr_2021_113930 crossref_primary_10_1016_j_ejphar_2025_177786 crossref_primary_10_1038_s41589_025_01874_8 crossref_primary_10_1002_wsbm_1515 crossref_primary_10_3390_jdb12010001 crossref_primary_10_1016_j_tips_2022_01_008 crossref_primary_10_1016_j_ijpharm_2024_124344 crossref_primary_10_1002_ange_202003545 crossref_primary_10_1002_wsbm_1630 crossref_primary_10_2147_CPAA_S418314 crossref_primary_10_3390_life14060668 crossref_primary_10_37521_ejpps30211 crossref_primary_10_1002_1873_3468_13659 crossref_primary_10_14348_molcells_2023_2165 crossref_primary_10_3390_cells10113250 crossref_primary_10_1016_j_cbpa_2024_102499 crossref_primary_10_1038_s41398_019_0660_x crossref_primary_10_1093_stmcls_sxac020 crossref_primary_10_3389_fcell_2022_901510 crossref_primary_10_1093_jnen_nlae053 crossref_primary_10_1038_s42003_022_04043_y crossref_primary_10_3390_biomedicines9121836 crossref_primary_10_1016_j_ymthe_2019_03_009 crossref_primary_10_1016_j_colsurfb_2021_111991 crossref_primary_10_3389_ebm_2024_10266 crossref_primary_10_1016_j_ymthe_2023_07_002 crossref_primary_10_3390_ijms242317006 crossref_primary_10_3389_fcell_2022_1050856 crossref_primary_10_1016_j_jconrel_2020_08_023 crossref_primary_10_3390_cells12101381 crossref_primary_10_3390_ijms21228685 crossref_primary_10_1007_s12274_024_6776_1 crossref_primary_10_3389_fmed_2024_1328474 crossref_primary_10_3390_ijms252212034 crossref_primary_10_1080_14712598_2024_2392307 crossref_primary_10_3389_fcell_2025_1593207 crossref_primary_10_1016_j_actbio_2021_06_020 crossref_primary_10_1038_s41578_020_0209_x crossref_primary_10_1016_j_medj_2022_10_003 crossref_primary_10_3390_ijms22158148 crossref_primary_10_1038_s41592_021_01090_x crossref_primary_10_1093_lifemedi_lnad027 crossref_primary_10_1186_s13287_023_03282_y crossref_primary_10_1186_s12864_025_11269_7 crossref_primary_10_1186_s13287_020_01814_4 crossref_primary_10_3390_biomedicines13040765 crossref_primary_10_1038_s41598_022_12807_z crossref_primary_10_3390_cells9020504 crossref_primary_10_3389_fcell_2024_1491282 crossref_primary_10_1016_j_biomaterials_2021_121273 crossref_primary_10_1016_j_apsb_2024_04_015 crossref_primary_10_3390_ijms25052680 crossref_primary_10_1002_anie_202003545 crossref_primary_10_3390_biomedicines10010107 crossref_primary_10_1080_17460441_2021_1935859 crossref_primary_10_1007_s40142_021_00199_x crossref_primary_10_3389_fnano_2025_1475969 |
| ContentType | Journal Article |
| Copyright | Copyright © 2018 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved. |
| Copyright_xml | – notice: Copyright © 2018 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved. |
| DBID | CGR CUY CVF ECM EIF NPM 7X8 |
| DOI | 10.1016/j.ymthe.2018.12.009 |
| DatabaseName | Medline MEDLINE MEDLINE (Ovid) MEDLINE MEDLINE PubMed MEDLINE - Academic |
| DatabaseTitle | MEDLINE Medline Complete MEDLINE with Full Text PubMed MEDLINE (Ovid) MEDLINE - Academic |
| DatabaseTitleList | MEDLINE MEDLINE - Academic |
| Database_xml | – sequence: 1 dbid: NPM name: PubMed url: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=PubMed sourceTypes: Index Database – sequence: 2 dbid: 7X8 name: MEDLINE - Academic url: https://search.proquest.com/medline sourceTypes: Aggregation Database |
| DeliveryMethod | no_fulltext_linktorsrc |
| Discipline | Medicine Biology |
| EISSN | 1525-0024 |
| ExternalDocumentID | 30598301 |
| Genre | Journal Article Review |
| GroupedDBID | --- 0R~ 123 29M 2WC 36B 39C 4.4 53G 7X7 8FE 8FH AACTN AAEDW AALRI AAMRU AAVLU AAXUO ABJNI ABMAC ABUDA ACGFO ACGFS ACPRK ADBBV ADFRT ADVLN AENEX AFTJW AGAYW AHMBA AITUG AKAPO AKRWK ALIPV ALMA_UNASSIGNED_HOLDINGS AMRAJ AOIJS ASPBG AVWKF AZFZN BAWUL BBNVY BENPR BPHCQ BVXVI CGR CS3 CUY CVF DIK DU5 E3Z EBS ECM EIF EJD F5P FDB FEDTE FRP FYUFA GX1 HVGLF HYE HZ~ JIG KQ8 LG5 LK8 M41 M7P NPM O9- OK1 P2P PROAC RCE RIG RNTTT RPM SSZ TR2 W2D 7X8 AAYWO ABDGV ACVFH ADCNI AEUPX AFPUW AIGII AKBMS AKYEP APXCP EFKBS |
| ID | FETCH-LOGICAL-c487t-cc54c9ff794b1200722ab0a31a30c953e1f0b0a35f1fc6523d10db0f8b406b942 |
| IEDL.DBID | 7X8 |
| ISICitedReferencesCount | 69 |
| ISICitedReferencesURI | http://www.webofscience.com/api/gateway?GWVersion=2&SrcApp=Summon&SrcAuth=ProQuest&DestLinkType=CitingArticles&DestApp=WOS_CPL&KeyUT=000464017500007&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| ISSN | 1525-0024 |
| IngestDate | Sun Nov 09 10:24:58 EST 2025 Wed Feb 19 02:30:22 EST 2025 |
| IsDoiOpenAccess | false |
| IsOpenAccess | true |
| IsPeerReviewed | true |
| IsScholarly | true |
| Issue | 4 |
| Keywords | iPSCs messenger RNA reprogramming modified RNA mRNA reprogramming synthetic mRNA cellular reprogramming induced pluripotent stem cells |
| Language | English |
| License | Copyright © 2018 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved. |
| LinkModel | DirectLink |
| MergedId | FETCHMERGED-LOGICAL-c487t-cc54c9ff794b1200722ab0a31a30c953e1f0b0a35f1fc6523d10db0f8b406b942 |
| Notes | ObjectType-Article-1 SourceType-Scholarly Journals-1 ObjectType-Feature-2 ObjectType-Review-3 content type line 23 |
| OpenAccessLink | http://www.cell.com/article/S1525001618305938/pdf |
| PMID | 30598301 |
| PQID | 2162493137 |
| PQPubID | 23479 |
| ParticipantIDs | proquest_miscellaneous_2162493137 pubmed_primary_30598301 |
| PublicationCentury | 2000 |
| PublicationDate | 2019-04-10 |
| PublicationDateYYYYMMDD | 2019-04-10 |
| PublicationDate_xml | – month: 04 year: 2019 text: 2019-04-10 day: 10 |
| PublicationDecade | 2010 |
| PublicationPlace | United States |
| PublicationPlace_xml | – name: United States |
| PublicationTitle | Molecular therapy |
| PublicationTitleAlternate | Mol Ther |
| PublicationYear | 2019 |
| SSID | ssj0011596 |
| Score | 2.5351388 |
| SecondaryResourceType | review_article |
| Snippet | The discovery that ordinary skin cells can be turned into pluripotent stem cells by the forced expression of defined factors has raised hopes that personalized... |
| SourceID | proquest pubmed |
| SourceType | Aggregation Database Index Database |
| StartPage | 729 |
| SubjectTerms | Cell Differentiation - genetics Cellular Reprogramming - genetics Fibroblasts - metabolism Genetic Vectors Humans Induced Pluripotent Stem Cells - metabolism Induced Pluripotent Stem Cells - transplantation Nucleosides - genetics RNA, Messenger - genetics RNA, Small Interfering - genetics Transfection |
| Title | mRNA-Based Genetic Reprogramming |
| URI | https://www.ncbi.nlm.nih.gov/pubmed/30598301 https://www.proquest.com/docview/2162493137 |
| Volume | 27 |
| WOSCitedRecordID | wos000464017500007&url=https%3A%2F%2Fcvtisr.summon.serialssolutions.com%2F%23%21%2Fsearch%3Fho%3Df%26include.ft.matches%3Dt%26l%3Dnull%26q%3D |
| hasFullText | |
| inHoldings | 1 |
| isFullTextHit | |
| isPrint | |
| link | http://cvtisr.summon.serialssolutions.com/2.0.0/link/0/eLvHCXMwpV1LS8NAEB7UqnjxUV_1RQSvi7ub55xExeKloYhCbyG7mwUPTautQv-9s5uU3kTwEghhIexO5vtmvskMwE1sbWoSqVhSCs2csscyFJKh4ilqItDIjR82keZ5NhrhsE24zdqyyqVP9I7aTLTLkd9KkVCkEIowvZt-MDc1yqmr7QiNdejQE3RWnY5WKgJBtf-7KJYxc2C07Drk67sWY2JYrrYr8_lA_gvH9FjT3_vvW-7Dbssyg_vGLA5graq7sNXMnVx0YXvQKuqHEIxf8nv2QFhmAteCmhYERMqbqq0x4doRvPWfXh-fWTs1gWkKPuZM6zjSLhOLkRIuEyllqXgZijLkGuOwEpa7-9gKqxOKQ43gRnGbKcJ2hZE8ho16UlenEKQV8T-JFo3rW6iqElFJNOSrsYzSiPfgerkLBVmlkxrKupp8zYrVPvTgpNnKYtq0zyjIw2BGfuXsD6vPYYdOyMs3gl9Ax9I3WV3Cpv6ev88-r_xx0zUfDn4AtMaw-w |
| linkProvider | ProQuest |
| openUrl | ctx_ver=Z39.88-2004&ctx_enc=info%3Aofi%2Fenc%3AUTF-8&rfr_id=info%3Asid%2Fsummon.serialssolutions.com&rft_val_fmt=info%3Aofi%2Ffmt%3Akev%3Amtx%3Ajournal&rft.genre=article&rft.atitle=mRNA-Based+Genetic+Reprogramming&rft.jtitle=Molecular+therapy&rft.au=Warren%2C+Luigi&rft.au=Lin%2C+Cory&rft.date=2019-04-10&rft.issn=1525-0024&rft.eissn=1525-0024&rft.volume=27&rft.issue=4&rft.spage=729&rft_id=info:doi/10.1016%2Fj.ymthe.2018.12.009&rft.externalDBID=NO_FULL_TEXT |
| thumbnail_l | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/lc.gif&issn=1525-0024&client=summon |
| thumbnail_m | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/mc.gif&issn=1525-0024&client=summon |
| thumbnail_s | http://covers-cdn.summon.serialssolutions.com/index.aspx?isbn=/sc.gif&issn=1525-0024&client=summon |