In vivo genome editing improves motor function and extends survival in a mouse model of ALS

Gaj, Thomas, Ojala, David S., Ekman, Freja K., Byrne, Leah C., Limsirichai, Prajit, Schaffer, David V.·2017·Science Advances
DOI 10.1126/sciadv.aar3952

Cited by (185)

Items 101–185 of 185

  1. Efficient Gene Expression in Human Stem Cell Derived-Cortical Organoids Using Adeno Associated Virus
    Ann‐Na Cho, Fiona Bright, Nicolle Morey, Carol G. Au, Lars Matthias Ittner, Yazi Diana Ke · 2022 · Cells
  2. CRISPR/Cas9 Technology as a Modern Genetic Manipulation Tool for Recapitulating of Neurodegenerative Disorders in Large Animal Models
    Mahdi Barazesh, Shiva Mohammadi, Yadollah Bahrami, Pooneh Mokarram, Mohammad Hossein Morowvat, Massoud Saidijam, Morteza Karimipoor, Soudabeh Kavousipour, Amir Reza Vosoughi, Korosh Khanaki · 2020 · Current Gene Therapy
  3. CRISPR-mediated rapid generation of neural cell-specific knockout mice facilitates research in neurophysiology and pathology
    Dan Xiao, Weifeng Zhang, Qing Wang, Xing Li, Yuan Zhang, Javad Rasouli, Giacomo Casella, Bogoljub Ćirić, Mark T. Curtis, Abdolmohamad M. Rostami et al. · 2021 · Molecular Therapy — Methods & Clinical Development
  4. Stem Cell Models and Gene Targeting for Human Motor Neuron Diseases
    Yashashree Karpe, Zhen-Yu Chen, Xue-Jun Li · 2021 · Pharmaceuticals
  5. Therapeutic potential of combined viral transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases
    Joshua Kuruvilla, Andrew Octavian Sasmita, Anna Pick Kiong Ling · 2018 · Neurological Sciences
  6. Implications of miRNAs dysregulation in amyotrophic lateral sclerosis: Challenging for clinical applications
    Yuka Mitsuhashi Koike, Osamu Onodera · 2023 · Frontiers in Neuroscience
  7. Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing
    Buhle Moyo, Lucas B.C. Brown, I. Khondaker, Gang Bao · 2025 · Biomaterials
  8. CRISPR-mediated gene correction links the ATP7A M1311V mutations with amyotrophic lateral sclerosis pathogenesis in one individual
    Yeomin Yun, Sung-Ah Hong, Ka-Kyung Kim, Daye Baek, Dongsu Lee, Ashwini M. Londhe, Minhyung Lee, Jihyeon Yu, Zachary T. McEachin, Gary Jonathan Bassell et al. · 2020 · Communications Biology
  9. The advances in CRISPR technology and 3D genome
    William Wang, Linlin Zhang, Xiangdong Wang, Yiming Zeng · 2018 · Seminars in Cell and Developmental Biology
  10. Vectors in CRISPR Gene Editing for Neurological Disorders: Challenges and Opportunities
    Kexin Xiong, Xiaxia Wang, Caicai Feng, Kaixiang Zhang, Di Chen, Sen Yang · 2025 · Advanced Biology
  11. Progress in and Prospects of Genome Editing Tools for Human Disease Model Development and Therapeutic Applications
    Hong Thi Lam Phan, Kyoungmi Kim, Ho Lee, Je Kyung Seong · 2023 · Genes
  12. Promising therapeutic aspects in human genetic imprinting disorders
    Yunqi Chao, Yifang Qin, Xinyi Zou, Xiangzhi Wang, Chenxi Hu, Fangling Xia, Chaochun Zou · 2022 · Clinical Epigenetics
  13. Advances in Treatments in Muscular Dystrophies and Motor Neuron Disorders
    Bhaskar Roy, Robert C. Griggs · 2020 · Neurologic Clinics
  14. Therapeutic tools for familial ALS
    William Camu, Elisa De La Cruz, Florence Esselin · 2022 · Revue Neurologique
  15. Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing
    Susana M. N. Simões, Miguel M. Lino, Angela Barrera, Catarina Rebelo, Francesca Tomatis, Andreia Vilaça, Christopher Breunig, Andrea M. Neuner, João Peça, Ricardo González et al. · 2024 · Angewandte Chemie International Edition
  16. Recent therapeutic advances in the treatment and management of amyotrophic lateral sclerosis: the era of regenerative medicine
    Fabiola De Marchi, Ivan Lombardi, Alessandro Bombaci, Luca Diamanti, Marco Olivero, Elisa Perciballi, Danilo Tornabene, Edvige Vulcano, Daniela Ferrari, Letizia Mazzini · 2025 · Expert Review of Neurotherapeutics
  17. A targeted approach: Gene and RNA editing for neurodegenerative disease treatment
    Chen‐Wei Huang, Wang-Zheqi Zhang, Yan Liao, Ting Hu, Jiamei Li, Changli Wang · 2025 · Life Sciences
  18. Vector enabled CRISPR gene editing – A revolutionary strategy for targeting the diversity of brain pathologies
    Helen Forgham, Liwei Liu, Jiayuan Zhu, Ibrahim Javed, Weibo Cai, Ruirui Qiao, Thomas Paul Davis · 2023 · Coordination Chemistry Reviews
  19. Increasing the precision of gene editing in vitro, ex vivo, and in vivo
    Katherine P. Mueller, Jared Carlson-Stevermer, Krishanu Saha · 2018 · Current Opinion in Biomedical Engineering
  20. Advancement in CRISPR/Cas9 Technology to Better Understand and Treat Neurological Disorders
    Aishika Datta, Deepaneeta Sarmah, Harpreet Kaur, Antra Chaudhary, Namrata Vadak, Anupom Borah, Sudhir V. Shah, Xin Wang, Pallab Bhattacharya · 2022 · Cellular and Molecular Neurobiology
  21. Pigs with an INS point mutation derived from zygotes electroporated with CRISPR/Cas9 and ssODN
    Fuminori Tanihara, Maki Hirata, Zhao Namula, Lanh Thi Kim, Naoaki Yoshimura, Qingyi Lin, Koki Takebayashi, Tetsushi Sakuma, Takashi Yamamoto, Takeshige Otoi · 2023 · Frontiers in Cell and Developmental Biology
  22. The CRIPSR /Cas gene‐editing system—an immature but useful toolkit for experimental and clinical medicine
    Yuyan Yang, Yue Huang · 2019 · Animal Models and Experimental Medicine
  23. CRISPR/Cas9 a genomic engineering technology for treatment in ALS mouse models
    Hamid Khan, Hammad Riaz, Adeel Ahmed, Mubin Mustafa Kiyani, Sahibzada Muhammad Jawad, Syed Shahab Ud Din Shah, Turki Abualait, Fawaz Al‐Hussain, Hongtao Li, Shahid Bashir · 2025 · Regenerative Therapy
  24. SOD1-targeting therapies for neurodegenerative diseases: a review of current findings and future potential
    John Franklin, Mimoun Azzouz, Pamela J. Shaw · 2020 · Expert Opinion on Orphan Drugs
  25. What did CRISPR-Cas9 accomplish in its first 10 years?
    Yehya Khlidj · 2023 · Biochemia Medica
  26. In vivo and ex vivo gene therapy for neurodegenerative diseases: a promise for disease modification
    Pouyan Ebrahimi, Elham Davoudi, Razieh Sadeghian, Amin Zaki Zadeh, Emran Razmi, Reza Heidari, Mohammad Hossein Morowvat, Issa Sadeghian · 2024 · Naunyn-Schmiedeberg s Archives of Pharmacology
  27. Significance of gene therapy in neurodegenerative diseases
    Lingling Wang, Lin Ma, Zihan Gao, Ying Wang, Jiaoxue Qiu · 2025 · Frontiers in Neuroscience
  28. Amyotrophic Lateral Sclerosis
    Robin Warner · 2020 · IntechOpen eBooks
  29. Therapeutic Approaches to Amyotrophic Lateral Sclerosis from the Lab to the Clinic
    Vivek P. Chavda, Chirag Amrutlal Patel, Dharti H. Modh, Yavuz Nuri Ertaş, Shreya S. Sonak, Nafesa K. Munshi, Krishnan Anand, Arun Soni, Sonal Vijay Pande · 2022 · Current Drug Metabolism
  30. Targeting Amyotrophic Lateral Sclerosis with Gene Therapy: From Silencing Genes to Enhancing Neuroprotection
    Sergi Verdés, Xavier Navarro, Assumpció Bosch · 2025 · Human Gene Therapy
  31. Neurodegenerative Diseases - Molecular Mechanisms and Current Therapeutic Approaches
    Nagehan Ersoy Tunalı · 2020 · IntechOpen eBooks
  32. Gene therapy of neurodegenerative diseases: achievements, developments, and clinical implementation challenges
    E. V. Melnikova, Вадим Анатольевич Меркулов, O. V. Merkulova · 2023 · Biological Products Prevention Diagnosis Treatment
  33. Canadian Regulatory Framework and Regulatory Requirements for Cell and Gene Therapy Products
    Jian Wang, Emily Griffiths, Omar Tounekti, Martin Nemec, Éric Deneault, Jessie R. Lavoie, Anthony A.G. Ridgway · 2023 · Advances in experimental medicine and biology
  34. GHR-mutant pig derived from domestic pig and microminipig hybrid zygotes using CRISPR/Cas9 system
    Fuminori Tanihara, Maki Hirata, Zhao Namula, Manita Wittayarat, Lanh Thi Kim, Qingyi Lin, Koki Takebayashi, Hiromasa Hara, Megumi Nagahara, Takeshige Otoi · 2023 · Molecular Biology Reports
  35. CRISPR/Cas9 mediated somatic gene therapy for insertional mutations: the vibrator mouse model
    Xin Fu, Jie Zhu, Yaou Duan, Paul Lu, Kang Zhang · 2021 · Precision Clinical Medicine
  36. CRISPR–Cas Gene Editing for Neurological Disease
    Bryan P. Simpson, Beverly L. Davidson · 2019 · Elsevier eBooks
  37. A bibliometric analysis of gene editing and amyotrophic lateral sclerosis (from 2004 to 2024)
    Hejia Wan, Wenli Qian, Bingqi Wei, Kaiyue Tian, Ziyi Chen, Jiong Zhang, Fang Chen · 2024 · Frontiers in Neuroscience
  38. Recent advances in CRISPR/Cas9-assisted gene therapy
    Apeksha Srivastava, Shikha Chauhan, Vishal Ahuja · 2023 · Karbala International Journal of Modern Science
  39. The Impact of Splicing Dysregulation on Neuromuscular Disorders and Current Neuromuscular Genetic Therapies
    Jing Wu, Shuxuan Yan, Yuqin Bian, Rui Liu, Xinxing Lyu, Zhaoxiang Zhang, Shuhong Huang, Tao Chen, Lin Cheng · 2025 · Journal of Neurochemistry
  40. CRISPR: a tool with potential for genomic reprogramming in neurological disorders
    Yogesh Kumar Dhuriya, Aijaz Ahmad Naik · 2022 · Molecular Biology Reports
  41. Genetic therapies for neurological diseases
    Ahad A. Rahim, Manju A. Kurian, Haiyan Zhou, Ross Ferguson, Sarah J. Tabrizi, Gabriele Lignani, Kristian Aquilina, Simon N. Waddington · 2025 · Pharmacological Reviews
  42. Cutting-edge treatments in amyotrophic lateral sclerosis: the role of molecular pathogenesis in targeted therapies
    Ramin Raoufinia, Ghazal Alyari, Amin Tadayoni Nia, Mohammad Reza Abbaszadegan, Ali Mahmoudi, Sajjad Shafaeibajestan, Ehsan Saburi, Jalil Tavakol‐Afshari, Mehdi Hassani, Faezeh Jamali et al. · 2025 · Stem Cell Research & Therapy
  43. Applications of genome editing technologies in the treatment of human diseases
    Jamal Alshorman, Mohammad Javad Mehran, Kingsley Miyanda Tembo, Niusha Mostafavi, Mansoor Bolideei, Y L Wang · 2026 · Gene Therapy
  44. Applications of CRISPR/CAS9 Technology in Mice and Livestock Genome Editing: Current Research
    Dorota Maj, Klaudiusz Górka · 2024 · Annals of Animal Science
  45. Targets and Gene Therapy of ALS (Part 1)
    O. A. Shiryaeva, Christina Tolochko, Tatiana M. Alekseeva, Vyacheslav A. Dyachuk · 2025 · International Journal of Molecular Sciences
  46. Manufacturing and Delivering Genome-Editing Proteins
    Jia Liu, Ya-jun Liang, Pei-ling Ren, Thomas Gaj · 2018 · Methods in molecular biology
  47. CRISPR-Cas9: bridging the gap between aging mechanisms and therapeutic advances in neurodegenerative disorders
    Anas Shamsi, Mohammed Alrouji, Othman AlOmeir, Syed Tasqeruddin, Dinislam Khuzin, Azna Zuberi · 2025 · Frontiers in Cellular Neuroscience
  48. A Translational Roadmap for Neurological Nonsense Mutation Disorders
    Jiaqing Li, Zhenyun Zhu, Sanqing Xu · 2026 · International Journal of Molecular Sciences
  49. Cancer Therapy with CRISPR/Cas9: Prospects and Challenges
    Muhammad Fahad Chaudhary, Unsa Saleem, Samia Khan, Rabiya Khan, Muhammad Asim, Fakhra Azam, Mariam Sharif, Zainab Zainab, Ali Abbas Dilawar, Muhammad Jehanzaib · 2021 · International Journal of Environment Agriculture and Biotechnology
  50. Gene editing technology for improving life quality: A dream coming true?
    Roya Ramezankhani, Neda Minaei, Mahnaz Haddadi, Shukoofeh Torabi, Mahdi Hesaraki, Hamed Mirzaei, Massoud Vosough, Catherine M. Verfaillie · 2020 · Clinical Genetics
  51. The Control and Trust of CRISPR / Cas9 Genome Altering for Clinical Application with Gene Treatment and Treatment of Hereditary Diseases
    Hossein Soltaninejad, Mohadeseh Khoshandam, Amir Ali Hamidieh, Saman Hosseinkhani · 2022
  52. All-in-one AAV-mediated Nrl gene inactivation rescues retinal degeneration in Pde6a mice
    Zhiquan Liu, Siyu Chen, Chien‐Hui Lo, Qing Kenneth Wang, Yang Sun · 2024 · JCI Insight
  53. Structural mechanisms and insights on multiple nanobodies binding diverse SOD1 epitopes
    Shihao Cheng, Chao Zhong, Haoran Zhu, Kunting Mu, He Jiang, Peiyu Zhong, Z. Ma, Xihuan Liu, Ziying Wang, Rui Liu et al. · 2025 · Communications Biology
  54. Prime editing of the β1 adrenoceptor in the brain restores physiological REM sleep in a mouse model of Alzheimer’s disease
    Desirée Böck, Lisa Tidecks, Maria Wilhelm, Waleed ElGrawani, Sian N. Duss, Chiara Trevisan, Dalila Laura Vena, Anna Maria Reuss, Lucas Kissling, Matteo Ranucci et al. · 2025 · Nature Communications
  55. Gene therapy for aging: Current evidence and future directions
    Y C Zhang, Lu Gan, Shixuan Wang, Jinjin Zhang · 2026 · Ageing Research Reviews
  56. In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression
    Bae-Geun Nam, Jiwon Seo, Sung-Ah Hong, Juhee Kim, Minju Kang, Geneva Rose Notario, Yoontaik Hong, Seunghee Cho, Sangsu Bae, Sung-Rae Cho · 2026 · Genome Medicine
  57. Research progress on non-viral vectors of CRISPR/Cas9 system
    Qi Liu, Zhanzhan Zhang, Yang Liu · 2019 · Scientia Sinica Chimica
  58. Advances in CRISPER/Cas system and genome editing technologies for the treatment of neurodegenerative diseases
    Shailendra Kumar Mishra, Hung‐Ming Chang, Ahmad A. Obaid, Sandeep Kumar Singh · 2024 · Elsevier eBooks
  59. Therapeutic Strategies in Neurodegenerative Diseases
    Abdulmunaim M. Eid, Alireza Faridar, Stanley H. Appel · 2024 · Springer eBooks
  60. New therapies for neuromuscular diseases in 2018
    Apeksha M Shah, Ammar Al‐Chalabi · 2018 · The Lancet Neurology
  61. DNA Editing for Amyotrophic Lateral Sclerosis: Leading Off First Base
    Thomas J. Cunningham, Elizabeth Fisher, Pietro Fratta, Jonathan D. Gilthorpe · 2020 · The CRISPR Journal
  62. Neuroprotection in Amyotrophic Lateral Sclerosis
    Kewal K. Jain · 2019 · Springer protocols handbooks/Springer protocols
  63. Single AAV-mediated scarless genome editing in dysfunctional retinal neurons mediates robust visual restoration in mice
    Koji M Nishiguchi, Kosuke Fujita, Shota Katayama, Toru Nakazawa · 2019 · bioRxiv (Cold Spring Harbor Laboratory)
  64. 2016 in Review and Message from the Editors to our Reviewers
    Stefan M. Pulst, Nicholas Elwood Johnson, Alexandra Dürr, Massimo Pandolfo, Raymond P. Roos, Jeffery M. Vance · 2017 · Neurology Genetics
  65. CRISPR Falling on Deaf Ears
    Gaétan Burgio · 2018 · The CRISPR Journal
  66. Molecular mechanism of amyotrophic lateral sclerosis (ALS) from the viewpoint of the formation and degeneration of transactive response DNA-binding protein 43 kDa (TDP-43) inclusions
    Sou Kasahara, Tomohiko Ishihara, Yuka Mitsuhashi Koike, Akihiro Sugai, Osamu Onodera · 2020 · Rinsho Shinkeigaku
  67. Gene editing and central nervous system regeneration
    Sara Monteiro Lopes, Luís Pereira de Almeida · 2020 · Elsevier eBooks
  68. 2018: Year in Review and Message from the Editors to Our Reviewers
    Stefan M. Pulst, Raymond P. Roos, Alexandra Dürr, Jeffery M. Vance, Margherita Milone, Massimo Pandolfo · 2019 · Neurology Genetics
  69. Development of transposon-mediated gene delivery systems with improved safety for the use in gene and cell therapy
    Maximilian Amberger · 2023
  70. Near‐Infrared Light Activated Formulation for the Spatially Controlled Release of CRISPR‐Cas9 Ribonucleoprotein for Brain Gene Editing
    Susana M. N. Simões, Miguel M. Lino, Angela Barrera, Catarina Rebelo, Francesca Tomatis, Andreia Vilaça, Christopher Breunig, Andrea M. Neuner, João Peça, Ricardo González et al. · 2024 · Angewandte Chemie
  71. CRISPR/Cas9-based genetic engineering for translational research in neurological disorders
    Platon Megagiannis, Guy Armand Rouleau, Yang Zhou · 2024 · Elsevier eBooks
  72. Gene therapy under clinical trials for neurodegenerative diseases
    Bhupender Sahu, Lakshita Tyagi, Prshant Tyagi, Umesh Kumar · 2024 · Elsevier eBooks
  73. Therapeutic potential of genome editing tools in neurodegenerative diseases
    Ankur Kumar, Nandigam Pardhipa, Sachin G. Swami, Vibha Dwivedi, Anand Kamal Singh · 2024 · Elsevier eBooks
  74. Advancement of CRISPR/Cas9 in Translational Biomedicines
    Neha Atale, Qinglin Kong, Vibha Rani · 2025 · Advances in Science, Technology & Innovation/Advances in science, technology & innovation
  75. Pre-clinical evaluation of a gene therapy candidate for SOD1 -ALS shows improved survival and signs of inflammation in the CNS of treated mice
    Sonia Pezet, Jennifer Hua, Thibaut Marais, Marine Delamare, Sahar Elouej, Julia P. Lemos, Alexia Castiglione, Stéphanie Astord, Mathilde Cohen-Tannoudji, Georges Arielle Pêche et al. · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
  76. Physiological and pathological roles of ANXA11: a multifunctional regulator in neurodegeneration and other disorders
    Chenyang Liu, Yongting Lu, Honglin Zheng, Suying Duan, Heng Zhang, Yaochong Zhang, Liu Han, Haiyang Luo, Yuming Xu · 2026 · Cell Communication and Signaling
  77. HML-2 env knockdown by AAV9-mediated miRNAs attenuates amyotrophic lateral sclerosis-like manifestations in mice
    Myoung-Hwa Lee, W. H. Li, Kevon Sampson, Stephanie H. Choi, Melina Jones, Joseph P. Steiner, Avindra Nath · 2026 · Brain
  78. Next-Generation Therapies for Genetic Diseases: The Synergy of CRISPR and Gene Therapy
    Pawan N. Karwa, Atharva Kharul, Vaibhav Parekar, Sonali Labhade, Nikhil S. Sakle, Ritesh P. Bhole, Harshad S. Kapare · 2026 · Biochemical Genetics
  79. Preclinical Models of Neurodegenerative Diseases
    Jan Cendelín, Jan Tuma · 2026 · Contemporary clinical neuroscience
  80. Current perspectives on the genetics of neurodegenerative diseases: A narrative review
    Shiva Chaudhary, Shreyas Iyer, Sonia Narwal, Meghnaa Mitra, Meghana Tare · 2026 · Aging advances.
  81. Exploring Genetic Therapies Targeting Amyotrophic Lateral Sclerosis in Animal Models: A Systematic Review and Meta‐Analysis
    Hannah E. Wedgwood, Richard I. Tuxworth, Zubair Ahmed · 2026 · The Journal of Gene Medicine
  82. CRISPR/Cas9-based nanodelivery systems for precision gene editing in neuroinflammation
    Sanjana Sahu, Parag Jain, Karan Kumar Dharme, Krishna Yadav · 2026 · Inflammation Research
  83. The Role of SOD1 and Oxidative Stress in Amyotrophic Lateral Sclerosis: An Overview
    Carlos Alberto Gómez-Pérez, Miguel Ángel Ramírez-García, Petra Yescas · 2026 · International Journal of Molecular Sciences
  84. Advancing the Translational Pipeline: From In Vivo and In Vitro 2D Models to Next-Generation Cell Culture Models for Amyotrophic Lateral Sclerosis Modelling and Drug Discovery
    Elisabetta Carata, Stefania Mariano, Bernardetta Anna Tenuzzo, Elisa Panzarini · 2026 · Biomedicines
  85. Unfolded Protein Response Pathway in Neurodegenerative Diseases: Genetic Insights into the Mechanisms Linking UPR Dysregulation to Tau Pathology and Therapeutic Implications
    Babu Swathy, Katherine S. LeSourd, Shauna H. Yuan · 2026 · Pharmacological Research