Published August 26, 2021 | Version v1
Publication Open

Understanding the Potential of Genome Editing in Parkinson's Disease

Creators

  • 1. Universidad de Los Andes

Description

CRISPR is a simple and cost-efficient gene-editing technique that has become increasingly popular over the last decades. Various CRISPR/Cas-based applications have been developed to introduce changes in the genome and alter gene expression in diverse systems and tissues. These novel gene-editing techniques are particularly promising for investigating and treating neurodegenerative diseases, including Parkinson's disease, for which we currently lack efficient disease-modifying treatment options. Gene therapy could thus provide treatment alternatives, revolutionizing our ability to treat this disease. Here, we review our current knowledge on the genetic basis of Parkinson's disease to highlight the main biological pathways that become disrupted in Parkinson's disease and their potential as gene therapy targets. Next, we perform a comprehensive review of novel delivery vehicles available for gene-editing applications, critical for their successful application in both innovative research and potential therapies. Finally, we review the latest developments in CRISPR-based applications and gene therapies to understand and treat Parkinson's disease. We carefully examine their advantages and shortcomings for diverse gene-editing applications in the brain, highlighting promising avenues for future research.

⚠️ This is an automatic machine translation with an accuracy of 90-95%

Translated Description (Arabic)

CRISPR هي تقنية بسيطة وفعالة من حيث التكلفة لتحرير الجينات أصبحت شائعة بشكل متزايد على مدى العقود الماضية. تم تطوير العديد من التطبيقات القائمة على كريسبر/كاس لإدخال تغييرات في الجينوم وتغيير التعبير الجيني في الأنظمة والأنسجة المتنوعة. تعد تقنيات تعديل الجينات الجديدة هذه واعدة بشكل خاص للتحقيق في الأمراض التنكسية العصبية وعلاجها، بما في ذلك مرض باركنسون، والتي نفتقر حاليًا إلى خيارات علاج فعالة لتعديل الأمراض. وبالتالي يمكن أن يوفر العلاج الجيني بدائل علاجية، مما يحدث ثورة في قدرتنا على علاج هذا المرض. هنا، نراجع معرفتنا الحالية على الأساس الجيني لمرض باركنسون لتسليط الضوء على المسارات البيولوجية الرئيسية التي تتعطل في مرض باركنسون وإمكاناتها كأهداف للعلاج الجيني. بعد ذلك، نجري مراجعة شاملة لمركبات التوصيل الجديدة المتاحة لتطبيقات تعديل الجينات، وهو أمر بالغ الأهمية لتطبيقها بنجاح في كل من الأبحاث المبتكرة والعلاجات المحتملة. أخيرًا، نستعرض أحدث التطورات في التطبيقات القائمة على كريسبر والعلاجات الجينية لفهم وعلاج مرض باركنسون. ندرس بعناية مزاياها وعيوبها لتطبيقات تحرير الجينات المتنوعة في الدماغ، مع تسليط الضوء على السبل الواعدة للبحث المستقبلي.

Translated Description (French)

CRISPR est une technique d'édition de gènes simple et rentable qui est devenue de plus en plus populaire au cours des dernières décennies. Diverses applications basées sur CRISPR/Cas ont été développées pour introduire des changements dans le génome et modifier l'expression des gènes dans divers systèmes et tissus. Ces nouvelles techniques d'édition de gènes sont particulièrement prometteuses pour étudier et traiter les maladies neurodégénératives, y compris la maladie de Parkinson, pour lesquelles nous manquons actuellement d'options de traitement efficaces pour modifier la maladie. La thérapie génique pourrait ainsi fournir des alternatives de traitement, révolutionnant notre capacité à traiter cette maladie. Ici, nous passons en revue nos connaissances actuelles sur la base génétique de la maladie de Parkinson pour mettre en évidence les principales voies biologiques qui sont perturbées dans la maladie de Parkinson et leur potentiel en tant que cibles de thérapie génique. Ensuite, nous effectuons un examen complet des nouveaux vecteurs disponibles pour les applications d'édition de gènes, essentiels à leur application réussie à la fois dans la recherche innovante et dans les thérapies potentielles. Enfin, nous passons en revue les derniers développements dans les applications basées sur CRISPR et les thérapies géniques pour comprendre et traiter la maladie de Parkinson. Nous examinons attentivement leurs avantages et leurs inconvénients pour diverses applications d'édition de gènes dans le cerveau, mettant en évidence des pistes prometteuses pour la recherche future.

Translated Description (Spanish)

CRISPR es una técnica de edición de genes simple y rentable que se ha vuelto cada vez más popular en las últimas décadas. Se han desarrollado varias aplicaciones basadas en CRISPR/Cas para introducir cambios en el genoma y alterar la expresión génica en diversos sistemas y tejidos. Estas novedosas técnicas de edición de genes son particularmente prometedoras para investigar y tratar enfermedades neurodegenerativas, incluida la enfermedad de Parkinson, para las cuales actualmente carecemos de opciones de tratamiento eficientes que modifiquen la enfermedad. Por lo tanto, la terapia génica podría proporcionar alternativas de tratamiento, revolucionando nuestra capacidad para tratar esta enfermedad. Aquí, revisamos nuestro conocimiento actual sobre la base genética de la enfermedad de Parkinson para resaltar las principales vías biológicas que se interrumpen en la enfermedad de Parkinson y su potencial como dianas de terapia génica. A continuación, realizamos una revisión exhaustiva de los nuevos vehículos de entrega disponibles para aplicaciones de edición de genes, fundamentales para su aplicación exitosa tanto en investigaciones innovadoras como en posibles terapias. Finalmente, revisamos los últimos desarrollos en aplicaciones basadas en CRISPR y terapias génicas para comprender y tratar la enfermedad de Parkinson. Examinamos cuidadosamente sus ventajas y deficiencias para diversas aplicaciones de edición de genes en el cerebro, destacando vías prometedoras para futuras investigaciones.

Files

pdf.pdf

Files (1.9 MB)

⚠️ Please wait a few minutes before your translated files are ready ⚠️ Note: Some files might be protected thus translations might not work.
Name Size Download all
md5:04edb4dabd2278ae6e349a9be96034ee
1.9 MB
Preview Download

Additional details

Additional titles

Translated title (Arabic)
فهم إمكانات تعديل الجينوم في مرض باركنسون
Translated title (French)
Comprendre le potentiel de l'édition du génome dans la maladie de Parkinson
Translated title (Spanish)
Comprender el potencial de la edición del genoma en la enfermedad de Parkinson

Identifiers

Other
https://openalex.org/W3193582159
DOI
10.3390/ijms22179241

Is supplement to
https://openalex.org/W2960163646 (Other)
https://openalex.org/W2903635918 (Other)
https://openalex.org/W2807464733 (Other)
https://openalex.org/W2667943050 (Other)
https://openalex.org/W2603161682 (Other)
https://openalex.org/W2516842346 (Other)
https://openalex.org/W2437118042 (Other)
https://openalex.org/W2357872089 (Other)
https://openalex.org/W2337751047 (Other)
https://openalex.org/W2134268326 (Other)

GreSIS Basics Section

Is Global South Knowledge
Yes
Country
Colombia

References

  • https://openalex.org/W1494864007
  • https://openalex.org/W1507910610
  • https://openalex.org/W1548404074
  • https://openalex.org/W1561899360
  • https://openalex.org/W1592617873
  • https://openalex.org/W1601161972
  • https://openalex.org/W167499259
  • https://openalex.org/W1709867787
  • https://openalex.org/W1710257910
  • https://openalex.org/W1762178123
  • https://openalex.org/W1795898662
  • https://openalex.org/W1858544150
  • https://openalex.org/W1873651958
  • https://openalex.org/W1947637767
  • https://openalex.org/W1962249777
  • https://openalex.org/W1965938007
  • https://openalex.org/W1966513242
  • https://openalex.org/W1967111894
  • https://openalex.org/W1971208141
  • https://openalex.org/W1971258758
  • https://openalex.org/W1972476556
  • https://openalex.org/W1972601497
  • https://openalex.org/W1977376243
  • https://openalex.org/W1977709885
  • https://openalex.org/W1981405716
  • https://openalex.org/W1982111205
  • https://openalex.org/W1988111175
  • https://openalex.org/W1989230986
  • https://openalex.org/W1991208841
  • https://openalex.org/W1992698841
  • https://openalex.org/W1994444930
  • https://openalex.org/W1996001296
  • https://openalex.org/W1996453271
  • https://openalex.org/W1999510776
  • https://openalex.org/W2000118489
  • https://openalex.org/W2003582994
  • https://openalex.org/W2005734278
  • https://openalex.org/W2006260503
  • https://openalex.org/W2014734622
  • https://openalex.org/W2017621114
  • https://openalex.org/W2019375221
  • https://openalex.org/W2019695510
  • https://openalex.org/W2020747873
  • https://openalex.org/W2023285404
  • https://openalex.org/W2023572125
  • https://openalex.org/W2024891592
  • https://openalex.org/W2027096397
  • https://openalex.org/W2028023684
  • https://openalex.org/W2030385272
  • https://openalex.org/W2034518767
  • https://openalex.org/W2037288337
  • https://openalex.org/W2039340367
  • https://openalex.org/W2039689914
  • https://openalex.org/W2041676728
  • https://openalex.org/W2044656113
  • https://openalex.org/W2044866051
  • https://openalex.org/W2050480410
  • https://openalex.org/W2054575697
  • https://openalex.org/W2056512830
  • https://openalex.org/W2060526494
  • https://openalex.org/W2061650108
  • https://openalex.org/W2061995199
  • https://openalex.org/W2064016373
  • https://openalex.org/W2064093384
  • https://openalex.org/W2065259720
  • https://openalex.org/W2065275335
  • https://openalex.org/W2068791841
  • https://openalex.org/W2073217307
  • https://openalex.org/W2074761817
  • https://openalex.org/W2075997145
  • https://openalex.org/W2076226743
  • https://openalex.org/W2076969496
  • https://openalex.org/W2080470197
  • https://openalex.org/W2081113434
  • https://openalex.org/W2082425146
  • https://openalex.org/W2086824808
  • https://openalex.org/W2087032263
  • https://openalex.org/W2087959569
  • https://openalex.org/W2088327714
  • https://openalex.org/W2088474579
  • https://openalex.org/W2089319449
  • https://openalex.org/W2090530492
  • https://openalex.org/W2091110313
  • https://openalex.org/W2091546373
  • https://openalex.org/W2092420017
  • https://openalex.org/W2093534313
  • https://openalex.org/W2094186182
  • https://openalex.org/W2095401453
  • https://openalex.org/W2096298540
  • https://openalex.org/W2098220259
  • https://openalex.org/W2099610218
  • https://openalex.org/W2103540918
  • https://openalex.org/W2104722080
  • https://openalex.org/W2104965614
  • https://openalex.org/W2106350754
  • https://openalex.org/W2107910518
  • https://openalex.org/W2108038398
  • https://openalex.org/W2108724455
  • https://openalex.org/W2108865709
  • https://openalex.org/W2110664937
  • https://openalex.org/W2111837903
  • https://openalex.org/W2112709886
  • https://openalex.org/W2113188427
  • https://openalex.org/W2114294613
  • https://openalex.org/W2114741749
  • https://openalex.org/W2115603949
  • https://openalex.org/W2116534141
  • https://openalex.org/W2117065100
  • https://openalex.org/W2117937193
  • https://openalex.org/W2118598201
  • https://openalex.org/W2124668406
  • https://openalex.org/W2125987139
  • https://openalex.org/W2127038376
  • https://openalex.org/W2129665511
  • https://openalex.org/W2129676131
  • https://openalex.org/W2133057192
  • https://openalex.org/W2135606077
  • https://openalex.org/W2135907949
  • https://openalex.org/W2136230100
  • https://openalex.org/W2138811343
  • https://openalex.org/W2139647754
  • https://openalex.org/W2141355937
  • https://openalex.org/W2141570423
  • https://openalex.org/W2144605372
  • https://openalex.org/W2151844149
  • https://openalex.org/W2152330309
  • https://openalex.org/W2152569432
  • https://openalex.org/W2153694555
  • https://openalex.org/W2154737306
  • https://openalex.org/W2156202012
  • https://openalex.org/W2156806006
  • https://openalex.org/W2157645703
  • https://openalex.org/W2159400623
  • https://openalex.org/W2160051211
  • https://openalex.org/W2160854976
  • https://openalex.org/W2161788422
  • https://openalex.org/W2162730195
  • https://openalex.org/W2169373177
  • https://openalex.org/W2172425918
  • https://openalex.org/W2173249787
  • https://openalex.org/W2173737475
  • https://openalex.org/W2174314139
  • https://openalex.org/W2176154815
  • https://openalex.org/W2186612922
  • https://openalex.org/W2192049920
  • https://openalex.org/W2203047261
  • https://openalex.org/W2209187832
  • https://openalex.org/W2224398381
  • https://openalex.org/W2233300840
  • https://openalex.org/W2265120954
  • https://openalex.org/W2282819021
  • https://openalex.org/W2283197772
  • https://openalex.org/W2294256859
  • https://openalex.org/W2296911202
  • https://openalex.org/W2335249448
  • https://openalex.org/W2337871602
  • https://openalex.org/W2340446708
  • https://openalex.org/W2345399459
  • https://openalex.org/W2345889524
  • https://openalex.org/W2413468039
  • https://openalex.org/W2415670156
  • https://openalex.org/W2417922500
  • https://openalex.org/W2418253328
  • https://openalex.org/W2461013690
  • https://openalex.org/W2472509986
  • https://openalex.org/W2496542349
  • https://openalex.org/W2503377259
  • https://openalex.org/W2507157990
  • https://openalex.org/W2509318050
  • https://openalex.org/W2511725062
  • https://openalex.org/W2521004562
  • https://openalex.org/W2528153557
  • https://openalex.org/W2528854795
  • https://openalex.org/W2542840147
  • https://openalex.org/W2545511601
  • https://openalex.org/W2550769902
  • https://openalex.org/W2552166386
  • https://openalex.org/W2552600365
  • https://openalex.org/W2553051332
  • https://openalex.org/W2562386484
  • https://openalex.org/W2562658921
  • https://openalex.org/W2581151188
  • https://openalex.org/W2585272077
  • https://openalex.org/W2586717174
  • https://openalex.org/W2588858201
  • https://openalex.org/W2589507128
  • https://openalex.org/W2591817710
  • https://openalex.org/W2592146264
  • https://openalex.org/W2593151357
  • https://openalex.org/W2593342777
  • https://openalex.org/W2593974461
  • https://openalex.org/W2600387426
  • https://openalex.org/W2605903025
  • https://openalex.org/W2614291262
  • https://openalex.org/W2615449285
  • https://openalex.org/W2620315774
  • https://openalex.org/W2625047539
  • https://openalex.org/W2667943050
  • https://openalex.org/W2733906300
  • https://openalex.org/W2739933996
  • https://openalex.org/W2740431289
  • https://openalex.org/W2745438578
  • https://openalex.org/W2752243640
  • https://openalex.org/W2754278602
  • https://openalex.org/W2764213443
  • https://openalex.org/W2765809583
  • https://openalex.org/W2766599608
  • https://openalex.org/W2768640554
  • https://openalex.org/W2769601563
  • https://openalex.org/W2770443931
  • https://openalex.org/W2778181726
  • https://openalex.org/W2779247349
  • https://openalex.org/W2781514432
  • https://openalex.org/W2785277056
  • https://openalex.org/W2787206763
  • https://openalex.org/W2788036162
  • https://openalex.org/W2789042638
  • https://openalex.org/W2790727932
  • https://openalex.org/W2791875139
  • https://openalex.org/W2792407616
  • https://openalex.org/W2794875845
  • https://openalex.org/W2796225745
  • https://openalex.org/W2799602489
  • https://openalex.org/W2799652277
  • https://openalex.org/W2802060782
  • https://openalex.org/W2802061036
  • https://openalex.org/W2802426789
  • https://openalex.org/W2804452569
  • https://openalex.org/W2804566227
  • https://openalex.org/W2805823081
  • https://openalex.org/W2806820493
  • https://openalex.org/W2809659377
  • https://openalex.org/W2810249184
  • https://openalex.org/W2888335178
  • https://openalex.org/W2891818439
  • https://openalex.org/W2892735968
  • https://openalex.org/W2894636673
  • https://openalex.org/W2898863138
  • https://openalex.org/W2899726955
  • https://openalex.org/W2902575030
  • https://openalex.org/W2905435570
  • https://openalex.org/W2905778655
  • https://openalex.org/W2906190864
  • https://openalex.org/W2906751241
  • https://openalex.org/W2908367010
  • https://openalex.org/W2908648691
  • https://openalex.org/W2909064040
  • https://openalex.org/W2911036111
  • https://openalex.org/W2912684492
  • https://openalex.org/W2916149712
  • https://openalex.org/W2916632122
  • https://openalex.org/W2917018165
  • https://openalex.org/W2921132745
  • https://openalex.org/W2921339884
  • https://openalex.org/W2921986557
  • https://openalex.org/W2936115703
  • https://openalex.org/W2937074474
  • https://openalex.org/W2945739890
  • https://openalex.org/W2947777875
  • https://openalex.org/W2948868528
  • https://openalex.org/W2949380898
  • https://openalex.org/W2951163658
  • https://openalex.org/W2953856578
  • https://openalex.org/W2955555890
  • https://openalex.org/W2955664250
  • https://openalex.org/W2959296638
  • https://openalex.org/W2965120578
  • https://openalex.org/W2966824380
  • https://openalex.org/W2968206433
  • https://openalex.org/W2968955936
  • https://openalex.org/W2969465898
  • https://openalex.org/W2970975955
  • https://openalex.org/W2971869292
  • https://openalex.org/W2973312556
  • https://openalex.org/W2975264888
  • https://openalex.org/W2976425815
  • https://openalex.org/W2978243962
  • https://openalex.org/W2978981832
  • https://openalex.org/W2981378884
  • https://openalex.org/W2981880817
  • https://openalex.org/W2982128103
  • https://openalex.org/W2986329263
  • https://openalex.org/W2988754526
  • https://openalex.org/W2989798457
  • https://openalex.org/W2990016411
  • https://openalex.org/W2990601242
  • https://openalex.org/W2991009389
  • https://openalex.org/W2993040769
  • https://openalex.org/W2996926827
  • https://openalex.org/W2997890461
  • https://openalex.org/W3000036467
  • https://openalex.org/W3003625465
  • https://openalex.org/W3006139820
  • https://openalex.org/W3009423576
  • https://openalex.org/W3010662272
  • https://openalex.org/W3011375666
  • https://openalex.org/W3011802680
  • https://openalex.org/W3012801913
  • https://openalex.org/W3015140168
  • https://openalex.org/W3015141182
  • https://openalex.org/W3015892555
  • https://openalex.org/W3016213906
  • https://openalex.org/W3016796815
  • https://openalex.org/W3022663353
  • https://openalex.org/W3022670067
  • https://openalex.org/W3022911398
  • https://openalex.org/W3025772497
  • https://openalex.org/W3029234273
  • https://openalex.org/W3034029360
  • https://openalex.org/W3035960414
  • https://openalex.org/W3038068331
  • https://openalex.org/W3038131081
  • https://openalex.org/W3041653147
  • https://openalex.org/W3045414962
  • https://openalex.org/W3045432343
  • https://openalex.org/W3045680995
  • https://openalex.org/W3046002536
  • https://openalex.org/W3049557897
  • https://openalex.org/W3052421888
  • https://openalex.org/W3085769931
  • https://openalex.org/W3086606797
  • https://openalex.org/W3093557064
  • https://openalex.org/W3095197864
  • https://openalex.org/W3111927179
  • https://openalex.org/W3112308706
  • https://openalex.org/W3118550692
  • https://openalex.org/W3134994575
  • https://openalex.org/W3139386940
  • https://openalex.org/W3157769326
  • https://openalex.org/W3158448602
  • https://openalex.org/W4211123396
  • https://openalex.org/W4230212633
  • https://openalex.org/W4232045797
  • https://openalex.org/W4295078575
  • https://openalex.org/W4298181679
  • https://openalex.org/W4379371205
  • https://openalex.org/W577556467
  • https://openalex.org/W626716516
  • https://openalex.org/W889893070