The Promise and the Hope of Gene Therapy

It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the aff...
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Autor*in:	Eleni Papanikolaou [verfasserIn] Andreas Bosio [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2021

Schlagwörter:	genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR

Übergeordnetes Werk:	In: Frontiers in Genome Editing - Frontiers Media S.A., 2020, 3(2021)
Übergeordnetes Werk:	volume:3 ; year:2021

Links:	Link aufrufen Link aufrufen Link aufrufen Journal toc

DOI / URN:	10.3389/fgeed.2021.618346

Katalog-ID:	DOAJ001011340

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520			\|a It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
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allfields	10.3389/fgeed.2021.618346 doi (DE-627)DOAJ001011340 (DE-599)DOAJ0150aea3236049339e98244cab643a0d DE-627 ger DE-627 rakwb eng TP248.13-248.65 QH426-470 Eleni Papanikolaou verfasserin aut The Promise and the Hope of Gene Therapy 2021 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice. genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR Biotechnology Genetics Eleni Papanikolaou verfasserin aut Andreas Bosio verfasserin aut In Frontiers in Genome Editing Frontiers Media S.A., 2020 3(2021) (DE-627)1695605101 (DE-600)3017800-9 26733439 nnns volume:3 year:2021 https://doi.org/10.3389/fgeed.2021.618346 kostenfrei https://doaj.org/article/0150aea3236049339e98244cab643a0d kostenfrei https://www.frontiersin.org/articles/10.3389/fgeed.2021.618346/full kostenfrei https://doaj.org/toc/2673-3439 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 3 2021
spelling	10.3389/fgeed.2021.618346 doi (DE-627)DOAJ001011340 (DE-599)DOAJ0150aea3236049339e98244cab643a0d DE-627 ger DE-627 rakwb eng TP248.13-248.65 QH426-470 Eleni Papanikolaou verfasserin aut The Promise and the Hope of Gene Therapy 2021 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice. genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR Biotechnology Genetics Eleni Papanikolaou verfasserin aut Andreas Bosio verfasserin aut In Frontiers in Genome Editing Frontiers Media S.A., 2020 3(2021) (DE-627)1695605101 (DE-600)3017800-9 26733439 nnns volume:3 year:2021 https://doi.org/10.3389/fgeed.2021.618346 kostenfrei https://doaj.org/article/0150aea3236049339e98244cab643a0d kostenfrei https://www.frontiersin.org/articles/10.3389/fgeed.2021.618346/full kostenfrei https://doaj.org/toc/2673-3439 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 3 2021
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allfieldsGer	10.3389/fgeed.2021.618346 doi (DE-627)DOAJ001011340 (DE-599)DOAJ0150aea3236049339e98244cab643a0d DE-627 ger DE-627 rakwb eng TP248.13-248.65 QH426-470 Eleni Papanikolaou verfasserin aut The Promise and the Hope of Gene Therapy 2021 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice. genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR Biotechnology Genetics Eleni Papanikolaou verfasserin aut Andreas Bosio verfasserin aut In Frontiers in Genome Editing Frontiers Media S.A., 2020 3(2021) (DE-627)1695605101 (DE-600)3017800-9 26733439 nnns volume:3 year:2021 https://doi.org/10.3389/fgeed.2021.618346 kostenfrei https://doaj.org/article/0150aea3236049339e98244cab643a0d kostenfrei https://www.frontiersin.org/articles/10.3389/fgeed.2021.618346/full kostenfrei https://doaj.org/toc/2673-3439 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 3 2021
allfieldsSound	10.3389/fgeed.2021.618346 doi (DE-627)DOAJ001011340 (DE-599)DOAJ0150aea3236049339e98244cab643a0d DE-627 ger DE-627 rakwb eng TP248.13-248.65 QH426-470 Eleni Papanikolaou verfasserin aut The Promise and the Hope of Gene Therapy 2021 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice. genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR Biotechnology Genetics Eleni Papanikolaou verfasserin aut Andreas Bosio verfasserin aut In Frontiers in Genome Editing Frontiers Media S.A., 2020 3(2021) (DE-627)1695605101 (DE-600)3017800-9 26733439 nnns volume:3 year:2021 https://doi.org/10.3389/fgeed.2021.618346 kostenfrei https://doaj.org/article/0150aea3236049339e98244cab643a0d kostenfrei https://www.frontiersin.org/articles/10.3389/fgeed.2021.618346/full kostenfrei https://doaj.org/toc/2673-3439 Journal toc kostenfrei GBV_USEFLAG_A SYSFLAG_A GBV_DOAJ SSG-OLC-PHA GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_70 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_206 GBV_ILN_213 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_2014 GBV_ILN_4012 GBV_ILN_4037 GBV_ILN_4112 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4249 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4700 AR 3 2021
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container_title	Frontiers in Genome Editing
authorswithroles_txt_mv	Eleni Papanikolaou @@aut@@ Andreas Bosio @@aut@@
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author	Eleni Papanikolaou
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topic_title	TP248.13-248.65 QH426-470 The Promise and the Hope of Gene Therapy genome editing hemopoietic stem cell retroviral vectors designer nucleases CRISPR
topic	misc TP248.13-248.65 misc QH426-470 misc genome editing misc hemopoietic stem cell misc retroviral vectors misc designer nucleases misc CRISPR misc Biotechnology misc Genetics
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title	The Promise and the Hope of Gene Therapy
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title_sort	promise and the hope of gene therapy
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title_auth	The Promise and the Hope of Gene Therapy
abstract	It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
abstractGer	It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
abstract_unstemmed	It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
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title_short	The Promise and the Hope of Gene Therapy
url	https://doi.org/10.3389/fgeed.2021.618346 https://doaj.org/article/0150aea3236049339e98244cab643a0d https://www.frontiersin.org/articles/10.3389/fgeed.2021.618346/full https://doaj.org/toc/2673-3439
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