Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Cited by (290)

Items 201–290 of 290

1. Revolutionizing Huntington’s Disease Treatment: Breakthroughs in AAV-Mediated Gene Therapy
   Pedram Moeini Gavgani, Mario García‐Domínguez · 2025 · Cells
2. Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy
   Cecilia Mei, Valentina Magliocca, Xin Chen, Keith Massey, Anai Gonzalez-Cordero, Steven James Gray, Marco Tartaglia, Enrico Bertini, Stefania Paola Corti, Claudia Compagnucci · 2025 · Frontiers in Cellular Neuroscience
3. Higher Transduction Efficiency of AAV5 to Neural Stem Cells and Immature Neurons in Gerbil Dentate Gyrus Compared to AAV2 and rh10
   Yoshihide Sehara, Yuka Hayashi, Kenji Ohba, Ryosuke Uchibori, Masashi Urabe, Ayumu Inutsuka, Kuniko Shimazaki, Kensuke Kawai, Hiroaki Mizukami · 2021 · Human Gene Therapy
4. Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of RPE65 Leber congenital amaurosis
   Yanbo Liu, Jingjie Tai, Chaofeng Yu, Dan Xu, Dan Xiao, Jijing Pang · 2024 · Frontiers in Medicine
5. RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro
   Natalia V. Klementieva, Evgenii A. Lunev, Anna A. Shmidt, Elizaveta M. Loseva, Irina M. Savchenko, Ekaterina A. Svetlova, Ivan I. Galkin, Anna V. Polikarpova, Evgeny V. Usachev, Svetlana G. Vassilieva et al. · 2024 · Nucleic Acid Therapeutics
6. RNA interference-mediated silencing of Kv7.2 in rat dorsal root ganglion neurons abolishes the anti-nociceptive effect of a selective channel opener
   Markus Valdor, Anke Wagner, Heike Fischer, Viola Röhrs, Wolfgang Schröder, Gregor Bahrenberg, André Welbers, Henry Fechner, Jens Kurreck, Thomas Tzschentke et al. · 2020 · Journal of Pharmacological and Toxicological Methods
7. Strict network analysis of evolutionary conserved and brain-expressed genes reveals new putative candidates implicated in Intellectual Disability and in Global Development Delay
   Rafael Mina Piergiorge, Ana Tereza Ribeiro de Vasconcelos, Márcia Mattos Gonçalves Pimentel, Cíntia Barros Santos-Rebouças · 2020 · The World Journal of Biological Psychiatry
8. Challenges in development and authorisation of gene therapy products
   Солдатов Александр Алексеевич, Ж. И. Авдеева, Горенков Дмитрий Витальевич, L. M. Khantimirova, S. G. Guseva, Вадим Анатольевич Меркулов · 2022 · Biological Products Prevention Diagnosis Treatment
9. Preclinical study of rAAV2-sTRAIL: pharmaceutical efficacy, biodistribution and safety in animals
   Qiang Ru, Wei Li, Xiaoshuang Wang, Simin Zhang, L Chen, Yaoyin Zhang, Ying Chun Ge, Yan Zu, Yanxin Liu, Dexian Zheng · 2017 · Cancer Gene Therapy
10. Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Disorders of Inherited and Non-Inherited Origin
    Indu G. Rajapaksha, Peter Angus, Chandana B. Herath · 2019 · IntechOpen eBooks
11. Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice
    Ophélie Vacca, Amel Saoudi, Mathilde Doisy, Xaysongkhame Phongsavanh, Olivier Le Coz, Cathy Nagy, Julia Kuzniar, Cyrille Vaillend, Aurélie Goyenvalle · 2025 · Molecular Therapy — Nucleic Acids
12. Gene therapy for Parkinson’s disease: current landscape, translational challenges, and future directions
    Vito Evola, Mayur S. Parmar · 2026 · Expert Review of Neurotherapeutics
13. Recombinant DNA technology and gene therapy
    Yuvaraj Sivamani, Subrahmanya Hegde, Ajay R. Bhat, Harshit Sajal, Sumitha Elayaperumal · 2024 · Elsevier eBooks
14. Programmable macromolecule-based RNA-targeting therapies to treat human neurological disorders
    Kathryn H. Morelli, Aaron A. Smargon, Gene Wei-Ming Yeo · 2023 · RNA
15. Vector Tropism
    Shih‐Heng Chen, Bo He, Sarwyn Singh, Negin P. Martin · 2023 · Neuromethods
16. Spotlight on plasticity-related genes: Current insights in health and disease
    Nicola Brandt, Franziska Köper, Jens Hausmann, Anja Ursula Bräuer · 2024 · Pharmacology & Therapeutics
17. Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons
    Florie Borel, Eric L. Adams, Christian Mueller · 2019 · Methods in molecular biology
18. Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
    Andrea Boitnott, Matthew Rioux, Xin Chen, Steven James Gray · 2025 · Journal of Visualized Experiments
19. Artificial miRNA-mediated reduction of SNCA for the treatment of α-synucleinopathies
    Bradford M. Elmer, Erik Wischhof, Tiffany Chan, Thangaaishwarya Mahendran, Jeffrey A. Ardinger, Brenda Richards, Dongyu Liu, John H. Bladon, Ye He, Robert Jackson et al. · 2025 · Molecular Therapy
20. 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
21. Recombinant adeno-associated virus mediated gene delivery in the extracranial nervous system of adult mice by direct nerve immersion
    Mette Richner, Nádia Pereira Gonçalves, Poul Henning Jensen, Jens Randel Nyengaard, Christian Bjerggaard Vægter, Asad Jan · 2022 · STAR Protocols
22. Iron Oxide Nanoparticles Application in Toxicity Therapeutics of CNS Disorders Indicated by Molecular MRI
    Lei Zhao, Tao Peng, Shawn Wu · 2025 · Nanotechnology and nanomaterials.
23. Developing a minimally invasive gene therapy for multiple sclerosis
    Paul J H Nijhuis, Maurits Romijn, Roy Honing, Giselle van Zon, Inge Huitinga, Fred de Winter, Joost Verhaagen · 2025 · Molecular Therapy — Methods & Clinical Development
24. Imaging pathological activities of human brain tissue in organotypic culture
    Etienne Savary · 2018 · Journal of Neurological Disorders
25. Crossing the Blood-Brain Barrier with AAVs: What’s After SMA?
    Yujia A. Chan, Benjamin E. Deverman · 2022 · AAPS advances in the pharmaceutical sciences series
26. AAV serotype PHP.eB achieves superior neuronal transduction efficiency compared to AAV9 in pigtail macaques following intracerebroventricular administration
    Michal G. Fortuna, Lyle H. Nyberg, Naz Taskin, Natalie Weed, Avery C. Hunker, Kathryn M. Gudsnuk, Melissa R. Berg, Boaz P. Levi, Ed S. Lein, Jonathan T. Ting · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
27. Receptor-Guided AAV Tropism Engineering via MATCH
    Nolan Graham, Satheesh Kumar, Joseph Rainaldi, Seoin Yang, Andrew J. Portell, Billy Santoso, Prashant Mali · 2026 · ACS Synthetic Biology
28. Evaluation of [18F]fluoroestradiol and ChRERα as a gene expression PET reporter system in rhesus monkey brain
    Bing Li, Palak Wadhwa, Walter Lerchner, Paolo Zanotti‐Fregonara, Jeih‐San Liow, Xuefeng Yan, Sami S. Zoghbi, Sridhar Goud Nerella, Sanjay Telu, Cheryl L. Morse et al. · 2024 · Molecular Therapy
29. Genome-Wide Genetic Screening in the Mammalian CNS
    Mary H. Wertz, Myriam Heiman · 2017 · Research and perspectives in neurosciences
30. Systemic Gene Delivery by Single-Dose Intracardiac Administration of scAAV2/9 and scAAV2/rh10 Variants in Newborn Rats
    Lucie Chansel‐Debordeaux, Mathieu Bourdenx, Nathalie Dutheil, Sandra Doveró, Marie‐Hélène Canron, Clément Jimenez, Erwan Bézard, Benjamin Dehay · 2018 · Human Gene Therapy Methods
31. A study of adeno-associated virus in cortical-thalamostriatal pathway
    Shuang Sun, Zhiwei Zhu, Tianqi He, Feiyu Chen, Xiaojun Wang, Xiao Zhang, Min Li, Yuchuan Li, Yue Sun, Qin He et al. · 2021 · Brain Research
32. Nanoparticle-Mediated Upconversion of Near-Infrared Light: A Step Closer to Optogenetic Neuromodulation in Humans
    David Bergeron · 2018 · Stereotactic and Functional Neurosurgery
33. In vivo high-throughput screening of novel adeno-associated viral capsids targeting adult neural stem cells in the subventricular zone
    Sascha Dehler, Lukas P. M. Kremer, Santiago Cerrizuela, Thomas Stiehl, Jonas Weinmann, Heike Abendroth, Susanne Kleber, Alexander Laure, Jihad El Andari, Simon Anders et al. · 2021 · bioRxiv (Cold Spring Harbor Laboratory)
34. Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo
    Han Zhang, Karen L. Kelly, Jonathan Lee, Dimas Echeverria, David Cooper, Rebecca Panwala, Zexiang Chen, Nicholas Gaston, Gregory A. Newby, Jun Xie et al. · 2023 · bioRxiv (Cold Spring Harbor Laboratory)
35. Gene therapy in neurodegenerative disorders
    Hossam Tharwat Ali, Idris Sula, Ahmed Elrefaey, Yara Ashour, Mennatullah Mohamed Eltaras, Haya Mohamed, Mostafa Mahmoud Meshref · 2024 · Elsevier eBooks
36. Gene therapy and genome-editing for schwannoma in NF2-related schwannomatosis: current understanding and future directions
    Ryota Tamura, Masahiro Yo, Masahiro Toda · 2025 · Journal of Neuro-Oncology
37. Recent advances in gene therapy for neurological disorders: A comprehensive overview
    Diksha Jindal · 2025 · Adesh University Journal of Medical Sciences & Research
38. Advances in Gene Therapy for CLN2 (Batten) Disease
    Bishnu P. De, Jonathan B. Rosenberg, Philip L. Leopold, Stephen M. Kaminsky, Dolan Sondhi, Ronald G. Crystal · 2025 · Springer eBooks
39. Administration of a barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport
    Yulia Dzhashiashvili, Jodi L. McBride, Emily B. Fabyanic, Xin Huang, Brian M. Kelly, Greglynn D Walton-Gibbs, Vimala Vemireddi, Joan R. Wicks, Mohamad Nayal, Ariel A. Hippen et al. · 2025 · Molecular Therapy
40. Targeted Gene and Genome-Editing Strategies for Epilepsy: Experimental Advances and Translational Challenges
    Mohd Yaqub Mir, Bilal Ahmad Seh, Kashf Rafiq, Ádám Légrádi · 2026 · International Journal of Molecular Sciences
41. Pharmacotherapies for Gaucher disease: what’s new on the horizon?
    Maya Bhattiprolu, Max Kappler, Tae‐Un Han, Mark Henderson, Yonatan Serlin, Ellen Sidransky · 2026 · Expert Opinion on Pharmacotherapy
42. Autonomous intranasal delivery systems for central nervous system therapeutics
    Haosheng Shen, Santosh Kumar Srivastava, Nikhil Aggarwal, Matthew Wook Chang · 2026 · Experimental & Molecular Medicine
43. Gene Therapy Blueprints for NeuroAIDS
    Hector E. Rodriguez, Seetha Lakshmi, Charurut Somboonwit, Asa Z. Oxner, Lucy Guerra, Anteneh Addisu, Louise Gutierrez, John T. Sinnott, Christina Nilofer, Pandjassarame Kangueane et al. · 2017 · Springer eBooks
44. Regenerative Medicine in the Digital Age
    Arthur André · 2018 · Health informatics
45. Huntingtin lowering therapeutics
    Neil Aronin M. D, Miguel Sena‐Esteves, Anastasia Khvorova, Marian DiFiglia, Michael Brodsky · 2024 · Elsevier eBooks
46. Efficient transduction of adeno-associated virus vectors into gerbil hippocampus with an appropriate combination of viral capsids and promoters
    Yoshihide Sehara, Kuniko Shimazaki, Fumio Kurosaki, Naoki Kaneko, Ryosuke Uchibori, Masashi Urabe, Kensuke Kawai, Hiroaki Mizukami · 2018 · Neuroscience Letters
47. THE JEREMIAH METZGER LECTURE NOVEL THERAPEUTIC STRATEGIES OF ALLERGIC AND IMMUNOLOGIC DISORDERS.
    Ronald G. Crystal, Odelya E. Pagovich · 2018 · Europe PMC (PubMed Central)
48. Viral Vectors for Studying Drug-Seeking Behavior
    Arlene Martínez-Rivera, Caitlin E. Burgdorf, Anjali M. Rajadhyaksha · 2020 · Neuromethods
49. Cell-Based Delivery Approaches for DNA-Binding Domains to the Central Nervous System
    Peter Deng, Julian A. N. M. Halmai, Jennifer J Waldo, Kyle D. Fink · 2021 · Current Neuropharmacology
50. Intravenous injection of AAV-PHP.eB across the blood-brain barrier in the adult mouse for central nervous system gene therapy*
    Yongwei Shu, Jie Yao, Yang Qu, Jing Zheng, Jing Ding, Lina Zhang, Yefan Wang, Linlin Zhao, Jingyu Zhang, Siqi Tang · 2019 · Oncology and Translational Medicine
51. Alzheimer’s disease: An overview of the current treatments
    Carolina Serrano Larrea, David Clavijo-Calderón · 2019 · Bionatura
52. Gene delivery for Parkinson's disease
    Piotr Chmielarz, Anne E. Panhelainen, Mikko Airavaara, Andrii Domanskyi · 2020 · Elsevier eBooks
53. Mechanisms to investigate the nervous system : genetic engineering with adeno-associated viruses
    Tess Torregrosa · 2020
54. Lysosomal disease
    Patrick B. Deegan, Timothy Martin Cox · 2020
55. Unlocking effective transgene expression potential in gene therapy with viral vectors
    Nur Shuhaidatul Sarmiza Abdul Halim, Noor Hanis Abu Halim, Lelamekala Vengidasan, Norashikin Zakaria, Ida Shazrina Ismail, Badrul Hisham Yahaya · 2021 · Biomedical Research and Therapy
56. Faculty Opinions recommendation of Adeno-associated virus vector as a platform for gene therapy delivery.
    Michael George Fehlings · 2020 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
57. Faculty Opinions recommendation of Presymptomatic training mitigates functional deficits in a mouse model of Rett syndrome.
    Santhosh Girirajan · 2021 · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
58. Regulation des Autophagieproteins ULK1 im MPTP-Mausmodell des Morbus Parkinson
    Dirk Balke · 2022
59. Bioengineering novel AAV9-mGULO-GT for multi-disease gene therapy: Targeting mutated GULO expression to cure scurvy and brain diseases.
    Jing Liu, Manish Kumar, Jing Guo, Luyuan Huang, Zhen Zhang, Yaofeng Wang, Rongping Luo, shilong chu, Shuyan Chen, Rongrong Liu et al. · 2023 · Research Square
60. Congenital Heart Malformations Masked by Infantile Gangliosidosis—Case Report and Growing Evidence for Metabolic Disease-Associated Aortopathies
    Dana Elena Mîndru, Elena Țarcă, Elena Emanuela Braha, Alexandrina-Ștefania Curpăn, Solange Tamara Roşu, Dana-Teodora Anton-Păduraru, Heidrun Adu­mi­­trăchioaiei, Valentin Bernic, Ioana Alexandra Pădureț, Alina-Costina Luca · 2024 · Diagnostics
61. Genetic Treatments for Rare Neurodegenerative Disorders
    Christina A. Kousparou · 2024 · IntechOpen eBooks
62. Biodistribution Analysis of Adeno-Associated Viral Vectors of Serotypes 9 and rh.10 Encoding Arylsulfatase A Following Prior Immunization with Serotype 9 in Pigs
    Aysilu I. Mullagulova, A. A. Shaimardanova, Yana Olegovna Mukhamedshina, Valeriya V. Solovyeva, Ahmad Ibrahim, Albert Anatolyevich Rizvanov · 2024 · Učënye zapiski Kazanskogo universiteta. Seriâ Estestvennye nauki/Učënye zapiski Kazanskogo universiteta. Seriâ Estestvennye nauki/Učenye zapiski Kazanskogo gosudarstvennogo universiteta. Seriâ Estestvennye nauki
63. Advances in Gene Therapy for Neurologic Disorders: An Overview
    Pallavi Chand, Trideva Sastri Koduru, Ashish Singh Chauhan, Souvik Chakraborty, Vikash Jakhmola · 2025 · BIO Integration
64. Advanced gene therapy in Parkinson’s disease through innovations in drug delivery systems
    Phuong-Anh Tran Nguyen, Thuy-vi Nguyen, Soyeun Park · 2025 · Journal of Pharmaceutical Investigation
65. Intra-arterial Gene Delivery for Neurodegenerative Diseases
    Jonathan M. Fischell · 2025 · Springer eBooks
66. AAV11 mediates efficient retrograde trans-neuronal transduction of astrocytes
    Nengsong Luo, Yunling Gao, Zengpeng Han, Fuqiang Xu, Kunzhang Lin · 2025 · bioRxiv (Cold Spring Harbor Laboratory)
67. Potential Implications of Advanced Macromolecule Drug Delivery Systems in the Therapeutic Management of Autism Spectrum Disorder
    Sonalika Bhalla, Rajnish Srivastava, Pradeepta Kumar Sarangi, Dhriti Verma, Ashok Kumar Sahoo · 2025 · Macromolecular Symposia
68. Additional file 2 of Granulocyte-colony stimulating factor gene therapy as a novel therapeutics for stroke in a mouse model
    Janet Menzie, Modi, Jigar, Hongyaun Xu, Bent, Andrew, Trujillo, Paula, Kristen Medley, Eugenia Jimenez, Shen, Jessica, Marshall, Michael, Rui Tao et al. · 2020 · Figshare
69. Additional file 3 of Granulocyte-colony stimulating factor gene therapy as a novel therapeutics for stroke in a mouse model
    Janet Menzie, Modi, Jigar, Hongyaun Xu, Bent, Andrew, Trujillo, Paula, Kristen Medley, Eugenia Jimenez, Shen, Jessica, Marshall, Michael, Rui Tao et al. · 2020 · Figshare
70. Additional file 1 of Granulocyte-colony stimulating factor gene therapy as a novel therapeutics for stroke in a mouse model
    Janet Menzie, Modi, Jigar, Hongyaun Xu, Bent, Andrew, Trujillo, Paula, Kristen Medley, Eugenia Jimenez, Shen, Jessica, Marshall, Michael, Rui Tao et al. · 2020 · Figshare
71. Discovery of Fluorinated Ultrapotent Chemogenetic Ligands for PET Imaging and Neural Modulation in Nonhuman Primates
    Sridhar Goud Nerella, Jeih‐San Liow, B. Li, Adrian E. S. Jenson, Matilah T. Pamie-George, Sami S. Zoghbi, Juan L. Gomez, Michael Michaelides, Chris Magnus, Scott M. Sternson et al. · 2026 · Journal of Medicinal Chemistry
72. Microphysiological systems to accelerate and de-risk AAV gene therapy development
    Patrizia Tornabene, Estelle Berreur, Adrian Roth, Magdalena J. Kasendra · 2026 · Advanced Drug Delivery Reviews
73. Characterization of rAAV vectors packaging in baculovirusinfected insect cells
    Magalie Penaud‐Budloo · 2018 · theses.fr (ABES)
74. Compréhension de la neurophysiopathologie de l'ataxie de Friedreich et développement d'une approche de thérapie génique dans un nouveau modèle murin
    Charline de Montigny · 2018 · theses.fr (ABES)
75. Thérapie génique pour l’amyotrophie spinale à l’aide de vecteur AAV
    Aurore Besse · 2019
76. Réponses immunitaires et induction de tolérance pour la thérapie génique rAAV du muscle basée sur le ciblage des hépatocytes : induction de tolérance et mécanismes immunitaires liés à la transduction des hépatocytes
    Laurent Bartolo · 2018 · theses.fr (ABES)
77. Les agrégats de PABPN1 dans la dystrophie musculaire oculopharyngée
    Fanny Roth · 2019 · theses.fr (ABES)
78. Syndrome de Rett et implication des organes périphériques : de la physiopathologie à la thérapie
    Emilie Borloz · 2021 · theses.fr (ABES)
79. Caractérisation de l’environnement thymique et de la thymopoïèse en conditions physiologiques et dans le contexte de stratégies thérapeutiques par ciblage direct du thymus
    Alice Machado · 2021
80. Preclinical gene therapy using recombinant AAV vectors in mouse models of two human diseases
    Yasemin Özgür Günes · 2022 · theses.fr (ABES)
81. Novel therapeutic strategies for pesticide-induced neurotoxicity
    P Padma Sree Vidya Devi, Prasann Kumar · 2026 · Elsevier eBooks
82. Whole-brain biodistribution of AAV2, AAV8, and AAV-B10 after unilateral intracerebroventricular injection
    Dezhuang Ye, Abhinav Kayithi, Jinyun Yuan, Isaac Wang, Tianqi Xu, Marc Boisvert, Marie-Ève Paquet, Dezhuang Ye · 2026 · Molecular Therapy Advances
83. Revolutionizing Neuroprotection
    Ahana Hazra, Rideb Chakraborty, Naureen Afrose, Pratibha Bhowmick, Mithun Bhowmick · 2026 · Wiley eBooks
84. Evolving Solutions for the Next Generation of Intrathecal Drug Delivery Systems
    Manuel Menéndez‐González · 2026 · Neuromethods
85. Biologic Therapies for Alleviating Neurodegeneration in Lysosomal Storage Diseases
    Liyan Qiu, Jonas Ungerbäck, Eric Bennett, Rasmus Otkjær Bak · 2026 · BioDrugs
86. Patient-derived tissue cultures complement neurospheres for preclinical evaluation of AAV-mediated gene delivery in glioblastoma
    Franziska Köhler, Katharina Heß, Cindy Koloske, Frank Gaunitz, Steffen Klaus Rosahl, Rüdiger Gerlach, Sonja Kallendrusch · 2026 · Journal of Neuro-Oncology
87. Hunter syndrome (mucopolysaccharidosis type II): Molecular mechanisms, CNS biomarkers, and emerging therapeutic strategies
    Amelia Michelle Sojung Moon, Hyung-Goo Kim, Sang Hoon Lee, Sang Hoon Kim, Euiyeon Lee, Mohammad Abdur Rashid, Yongkyu Park, Chong Kun Cheon, Mi-Hyeon Jang · 2026 · Experimental and Molecular Pathology
88. Adeno‐Associated Virus Vector‐Mediated Expression of Neuroligin 2 in the Mouse Hippocampus Attenuates the Progression of Pentylenetetrazol‐Induced Seizures
    Tomohiro Tokutake, Akane Hara, Ayano Murayama, Yuto Mitsuda, Jun Yokose, Yusuke Yano, Yuki Shigetsura, Mika Ito, Naomi Takino, Shinichi Kumagai et al. · 2026 · The Journal of Gene Medicine
89. AAV5-miHTT (AMT-130) gene therapy for Huntington's disease: A review of mechanisms and phase I/II clinical trial evidence
    Arpan Pathak, Monika Sharma, Ankit Pant, Aarushi Singh, Harsh Srivastava, Priya, Manoj Gadewar, Debashish Paramanick · 2026 · Gene Reports
90. Restoring Parkin function: an AAV gene therapy approach for early-onset Parkinson’s disease
    Sambuddha Basu, Tyler G. Demarest, Nicholas J. Gattone, Alexandra J Gilsrud, Brittany Wicks, Apeksha Khatiwada, Mohammad Nayal, Renee C. Gentzel, Daniel M. Cohen, Eric Wildon Kostuk et al. · 2026 · Frontiers in Molecular Neuroscience