Gene specific therapies – the next therapeutic milestone in neurology

Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending o...
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Autor*in:	Brenner, David [verfasserIn] Ludolph, Albert C. [verfasserIn] Weishaupt, Jochen H. [verfasserIn]

Format:	E-Artikel
Sprache:	Englisch

Erschienen:	2020

Übergeordnetes Werk:	Enthalten in: Neurological research and practice - [London] : BioMed Central, 2019, 2(2020), 1 vom: 08. Sept.
Übergeordnetes Werk:	volume:2 ; year:2020 ; number:1 ; day:08 ; month:09

Links:	Volltext

DOI / URN:	10.1186/s42466-020-00075-z

Katalog-ID:	SPR040891011

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allfields	10.1186/s42466-020-00075-z doi (DE-627)SPR040891011 (SPR)s42466-020-00075-z-e DE-627 ger DE-627 rakwb eng 610 ASE 610 ASE Brenner, David verfasserin aut Gene specific therapies – the next therapeutic milestone in neurology 2020 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges. Ludolph, Albert C. verfasserin aut Weishaupt, Jochen H. verfasserin aut Enthalten in Neurological research and practice [London] : BioMed Central, 2019 2(2020), 1 vom: 08. Sept. (DE-627)1038162947 (DE-600)2947493-0 2524-3489 nnns volume:2 year:2020 number:1 day:08 month:09 https://dx.doi.org/10.1186/s42466-020-00075-z kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER 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_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 2 2020 1 08 09
spelling	10.1186/s42466-020-00075-z doi (DE-627)SPR040891011 (SPR)s42466-020-00075-z-e DE-627 ger DE-627 rakwb eng 610 ASE 610 ASE Brenner, David verfasserin aut Gene specific therapies – the next therapeutic milestone in neurology 2020 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges. Ludolph, Albert C. verfasserin aut Weishaupt, Jochen H. verfasserin aut Enthalten in Neurological research and practice [London] : BioMed Central, 2019 2(2020), 1 vom: 08. Sept. (DE-627)1038162947 (DE-600)2947493-0 2524-3489 nnns volume:2 year:2020 number:1 day:08 month:09 https://dx.doi.org/10.1186/s42466-020-00075-z kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER 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_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 2 2020 1 08 09
allfields_unstemmed	10.1186/s42466-020-00075-z doi (DE-627)SPR040891011 (SPR)s42466-020-00075-z-e DE-627 ger DE-627 rakwb eng 610 ASE 610 ASE Brenner, David verfasserin aut Gene specific therapies – the next therapeutic milestone in neurology 2020 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges. Ludolph, Albert C. verfasserin aut Weishaupt, Jochen H. verfasserin aut Enthalten in Neurological research and practice [London] : BioMed Central, 2019 2(2020), 1 vom: 08. Sept. (DE-627)1038162947 (DE-600)2947493-0 2524-3489 nnns volume:2 year:2020 number:1 day:08 month:09 https://dx.doi.org/10.1186/s42466-020-00075-z kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER 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_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 2 2020 1 08 09
allfieldsGer	10.1186/s42466-020-00075-z doi (DE-627)SPR040891011 (SPR)s42466-020-00075-z-e DE-627 ger DE-627 rakwb eng 610 ASE 610 ASE Brenner, David verfasserin aut Gene specific therapies – the next therapeutic milestone in neurology 2020 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges. Ludolph, Albert C. verfasserin aut Weishaupt, Jochen H. verfasserin aut Enthalten in Neurological research and practice [London] : BioMed Central, 2019 2(2020), 1 vom: 08. Sept. (DE-627)1038162947 (DE-600)2947493-0 2524-3489 nnns volume:2 year:2020 number:1 day:08 month:09 https://dx.doi.org/10.1186/s42466-020-00075-z kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER 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_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 2 2020 1 08 09
allfieldsSound	10.1186/s42466-020-00075-z doi (DE-627)SPR040891011 (SPR)s42466-020-00075-z-e DE-627 ger DE-627 rakwb eng 610 ASE 610 ASE Brenner, David verfasserin aut Gene specific therapies – the next therapeutic milestone in neurology 2020 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges. Ludolph, Albert C. verfasserin aut Weishaupt, Jochen H. verfasserin aut Enthalten in Neurological research and practice [London] : BioMed Central, 2019 2(2020), 1 vom: 08. Sept. (DE-627)1038162947 (DE-600)2947493-0 2524-3489 nnns volume:2 year:2020 number:1 day:08 month:09 https://dx.doi.org/10.1186/s42466-020-00075-z kostenfrei Volltext GBV_USEFLAG_A SYSFLAG_A GBV_SPRINGER 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_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 2 2020 1 08 09
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source	Enthalten in Neurological research and practice 2(2020), 1 vom: 08. Sept. volume:2 year:2020 number:1 day:08 month:09
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author	Brenner, David
spellingShingle	Brenner, David ddc 610 Gene specific therapies – the next therapeutic milestone in neurology
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topic_title	610 ASE Gene specific therapies – the next therapeutic milestone in neurology
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title	Gene specific therapies – the next therapeutic milestone in neurology
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title_full	Gene specific therapies – the next therapeutic milestone in neurology
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author_browse	Brenner, David Ludolph, Albert C. Weishaupt, Jochen H.
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title_sort	gene specific therapies – the next therapeutic milestone in neurology
title_auth	Gene specific therapies – the next therapeutic milestone in neurology
abstract	Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges.
abstractGer	Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges.
abstract_unstemmed	Abstract Gene selective approaches that either correct a disease mutation or a pathogenic mechanism will fundamentally change the treatment of neurological disorders. Basically, gene specific therapies are designed to manipulate RNA expression or reconstitute gene expression and function depending on the disease mechanism. Considerable methodological advances in the last years have made successful clinical translation of gene selective approaches possible, based on RNA interference or viral gene reconstitution in spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and familial amyloid polyneuropathy (FAP). In this review, we provide an overview of the existing and coming gene specific therapies in neurology and discuss benefits, risks and challenges.
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title_short	Gene specific therapies – the next therapeutic milestone in neurology
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up_date	2024-07-03T18:54:50.750Z
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