Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Pfister, Edith L.; DiNardo, Natalie; Mondo, Erica; Borel, Florie; Conroy, Faith; Fraser, Cara; Gernoux, Gwladys; Han, Xin; Hu, Danjing; Johnson, Emily; Kennington, Lori; Liu, PengPeng; Reid, Suzanne J.; Sapp, Ellen; Vodicka, Petr; Kuchel, Tim; Morton, A. Jennifer; Howland, David; Moser, Richard; Sena-Esteves, Miguel; Gao, Guangping; Mueller, Christian; DiFiglia, Marian; Aronin, Neil

doi:10.1089/hum.2017.199

Cited by 96Genetic Neurodegenerative DiseasesRNA Interference and Gene DeliveryVirus-based gene therapy research

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

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Pfister, Edith L., DiNardo, Natalie, Mondo, Erica, Borel, Florie, Conroy, Faith, Fraser, Cara, Gernoux, Gwladys, Han, Xin +16 more·2018·Human Gene Therapy

DOI 10.1089/hum.2017.199PMC PMC6909722

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Abstract

Huntington's disease (HD) is a fatal neurodegenerative disease caused by a genetic expansion of the CAG repeat region in the huntingtin (HTT) gene. Studies in HD mouse models have shown that artificial miRNAs can reduce mutant HTT, but evidence for their effectiveness and safety in larger animals is lacking. HD transgenic sheep express the full-length human HTT with 73 CAG repeats. AAV9 was used to deliver unilaterally to HD sheep striatum an artificial miRNA targeting exon 48 of the human HTT mRNA under control of two alternative promoters: U6 or CβA. The treatment reduced human mutant (m) HTT mRNA and protein 50-80% in the striatum at 1 and 6 months post injection. Silencing was detectable in both the caudate and putamen. Levels of endogenous sheep HTT protein were not affected. There was no significant loss of neurons labeled by DARPP32 or NeuN at 6 months after treatment, and Iba1-positive microglia were detected at control levels. It is concluded that safe and effective silencing of human mHTT protein can be achieved and sustained in a large-animal brain by direct delivery of an AAV carrying an artificial miRNA.

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Multiple injections of AAV are required due to the limited distribution achieved with a single injection.
“However, multiple injections of AAV are required due to the limited distribution achieved with a single injection.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [43]
The Results (page unavailable) report that vector genomes were widely distributed in the neostriatum and that a single administration reduced mutant huntingtin mRNA in the caudate and putamen. Although the Discussion notes much lower vector levels in the cortex, the paper does not say multiple injections are required; its striatal findings run contrary to that claim. (LLM, conf=0.84)
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AAV delivered by intra-parenchymal injections efficiently silenced HTT in minipig, sheep, and NHP.
“AAV delivered by intra-parenchymal injections efficiently silenced HTT in minipig, sheep, and NHP 41, 43, 44.”
From A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system (2019) · cites this paper as [43]

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A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
MACDONALD, M. (1993) · Cell · doi:10.1016/0092-8674(93)90585-e · Cited by 8,366 · see citing works
A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.
Shah, M. A., Datson, N. A., Srinidhi, L., Stanton, V. P., MacDonald, M. E., Allard, M. W., Youngman, S., Am, F., Mott, R., Draths, K., et al. (1993) · Cell · Cited by 3,055 · see citing works
Microglia-specific localisation of a novel calcium binding protein, Iba1
Ito, D., Imai, Y., Ohsawa, K., Nakajima, K., Fukuuchi, Y., & Kohsaka, S. (1998) · Molecular Brain Research · doi:10.1016/s0169-328x(98)00040-0 · Cited by 1,476 · see citing works
Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Kordasiewicz, H. B., Stanek, L. M., Wancewicz, E. V., Mazur, C., McAlonis, M. M., Pytel, K. A., Artates, J. W., Weiss, A., Cheng, S. H., Shihabuddin, L. S., et al. (2012) · Neuron · doi:10.1016/j.neuron.2012.05.009 · Cited by 770 · see citing works
Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons
DiFiglia, M., Sapp, E., Chase, K., Schwarz, C., Meloni, A., Young, C., Martin, E., Vonsattel, J.-P., Carraway, R., Reeves, S. A., et al. (1995) · Neuron · doi:10.1016/0896-6273(95)90346-1 · Cited by 749 · see citing works
Self-citation
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
Harper, S. Q., Staber, P. D., He, X., Eliason, S. L., Martins, I. H., Mao, Q., Yang, L., Kotin, R. M., Paulson, H. L., & Davidson, B. L. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0501507102 · Cited by 682 · see citing works
HEAT repeats in the Huntington's disease protein
Andrade-Navarro, M. A., & Peer Bork, B. (1995) · Nature Genetics · doi:10.1038/ng1095-115 · Cited by 601 · see citing works
Analysis of Transduction Efficiency, Tropism and Axonal Transport of AAV Serotypes 1, 2, 5, 6, 8 and 9 in the Mouse Brain
Aschauer, D. F., Kreuz, S., & Rumpel, S. (2013) · PLoS ONE · doi:10.1371/journal.pone.0076310 · Cited by 522 · see citing works
Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits
DiFiglia, M., Sena‐Esteves, M., Chase, K. O., Sapp, E., Pfister, E., Sass, M., Yoder, J., Reeves, P., Pandey, R. K., Rajeev, K. G., et al. (2007) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0708285104 · Cited by 417 · see citing works
Self-citation
Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice
Boudreau, R. L., McBride, J. L., Martins, I., Shen, S., Xing, Y., Carter, B. J., & Davidson, B. L. (2009) · Molecular Therapy · doi:10.1038/mt.2009.17 · Cited by 339 · see citing works
Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
Rodríguez-Lebrón, E., Denovan‐Wright, E. M., Nash, K. R., Lewin, A. S., & Mandel, R. J. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.05.006 · Cited by 277 · see citing works
Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease
McBride, J. L., Pitzer, M. R., Boudreau, R. L., Dufour, B., Hobbs, T., Ojeda, S. R., & Davidson, B. L. (2011) · Molecular Therapy · doi:10.1038/mt.2011.219 · Cited by 247 · see citing works
Sustained effects of nonallele‐specific Huntingtin silencing
Drouet, V., Perrin, V., Hässig, R., Dufour, N., Aurégan, G., Alves, S., Bonvento, G., Brouillet, E., Luthi‐Carter, R., Hantraye, P., et al. (2009) · Annals of Neurology · doi:10.1002/ana.21569 · Cited by 217 · see citing works
Expression of shRNA From a Tissue-specific pol II Promoter Is an Effective and Safe RNAi Therapeutic
Giering, J. C., Grimm, D., Storm, T. A., & Kay, M. A. (2008) · Molecular Therapy · doi:10.1038/mt.2008.144 · Cited by 205 · see citing works
An ovine transgenic Huntington's disease model
Jacobsen, J. C., Bawden, C. S., Rudiger, S. R., McLaughlan, C. J., Reid, S. J., Waldvogel, H. J., MacDonald, M. E., Gusella, J. F., Walker, S. K., Kelly, J. M., et al. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq063 · Cited by 198 · see citing works
Self-citation
New anti-huntingtin monoclonal antibodies: implications for huntingtin conformation and its binding proteins
Ko, J., Ou, S. K.-H., & Patterson, P. H. (2001) · Brain Research Bulletin · doi:10.1016/s0361-9230(01)00599-8 · Cited by 191 · see citing works
Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
Cearley, C. N., Vandenberghe, L. H., Parente, M. K., Carnish, E. R., Wilson, J. M., & Wolfe, J. H. (2008) · Molecular Therapy · doi:10.1038/mt.2008.166 · Cited by 182 · see citing works
Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease
Stanek, L. M., Sardi, S. P., Mastis, B., Richards, A. R., Treleaven, C. M., Taksir, T. V., Misra, K., Cheng, S. H., & Shihabuddin, L. S. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.200 · Cited by 162 · see citing works
Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis
Wang, G., Liu, X., Gaertig, M. A., Li, S., & Li, X.-J. (2016) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1524575113 · Cited by 146 · see citing works
rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse
Machida, Y., Okada, T., Kurosawa, M., Oyama, F., Ozawa, K., & Nukina, N. (2006) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2006.02.141 · Cited by 127 · see citing works
Elimination of huntingtin in the adult mouse leads to progressive behavioral deficits, bilateral thalamic calcification, and altered brain iron homeostasis
Dietrich, P., Johnson, I. M., Alli, S., & Dragatsis, I. (2017) · PLoS Genetics · doi:10.1371/journal.pgen.1006846 · Cited by 103 · see citing works
Early and progressive circadian abnormalities in Huntington's disease sheep are unmasked by social environment
Morton, A. J., Rudiger, S. R., Wood, N. I., Sawiak, S. J., Brown, G. C., McLaughlan, C. J., Kuchel, T. R., Snell, R. G., Faull, R. L. M., & Bawden, C. S. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu047 · Cited by 90 · see citing works
Self-citation
Quantification Assays for Total and Polyglutamine-Expanded Huntingtin Proteins
Macdonald, D., Tessari, M. A., Boogaard, I., Smith, M. W., Pulli, K., Szynol, A., Albertus, F., Lamers, M. B. A. C., Dijkstra, S., Kordt, D., et al. (2014) · PLoS ONE · doi:10.1371/journal.pone.0096854 · Cited by 73 · see citing works
Further Molecular Characterisation of the OVT73 Transgenic Sheep Model of Huntington's Disease Identifies Cortical Aggregates
Reid, S. J., Patassini, S., Handley, R. R., Rudiger, S. R., McLaughlan, C. J., Osmand, A., Jacobsen, J. C., Morton, A. J., Weiss, A., Waldvogel, H. J., et al. (2013) · Journal of Huntington s Disease · doi:10.3233/jhd-130067 · Cited by 54 · see citing works
Self-citation
A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo
Coles, A. H., Osborn, M. F., Alterman, J. F., Turanov, A. A., Godinho, B. M. D. C., Kennington, L. A., Chase, K. O., D, N. A. M., & Khvorova, A. (2015) · Nucleic Acid Therapeutics · doi:10.1089/nat.2015.0578 · Cited by 38 · see citing works
Self-citation
Cellular Analysis of Silencing the Huntington’s Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140/Q140 Mice
Keeler, A. M., Sapp, E., Chase, K. O., Sottosanti, E., Danielson, E., Pfister, E. L., Stoica, L., DiFiglia, M., D, N. A. M., & Sena‐Esteves, M. (2016) · Journal of Huntington s Disease · doi:10.3233/jhd-160215 · Cited by 37 · see citing works
Self-citation
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Pfister, E. L., Chase, K. O., Sun, H., Kennington, L. A., Conroy, F., Johnson, E., Miller, R., Borel, F., D, N. A. M., & Mueller, C. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.04.011 · Cited by 30 · see citing works
Self-citation
Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing
Cambon, K., Zimmer, V., Martineau, S., Gaillard, M., Jarrige, M., Bugi, A., Miniarikova, J., Rey, M. J., Hässig, R., Dufour, N., et al. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.05.001 · Cited by 19 · see citing works
Frameless multimodal image guidance of localized convection-enhanced delivery of therapeutics in the brain
van der Bom, I. M. J., Moser, R. P., Gao, G., Sena‐Esteves, M., D, N. A. M., & Gounis, M. J. (2011) · Journal of NeuroInterventional Surgery · doi:10.1136/neurintsurg-2011-010170 · Cited by 16 · see citing works
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Finding the Striatum in Sheep: Use of a Multi-Modal Guided Approach for Convection Enhanced Delivery
van der Bom, I. M. J., Moser, R. P., Gao, G., Mondo, E., O'Connell, D. C., Gounis, M. J., McGowan, S., Chaurette, J., Bishop, N., Sena‐Esteves, M., et al. (2013) · Journal of Huntington s Disease · doi:10.3233/jhd-130053 · Cited by 12 · see citing works
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Hernaiz, A., Toivonen, J. M., Bolea, R., & Martín‐Burriel, I. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232012609 · Cited by 24 · see citing works
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Ghafouri‐Fard, S., Khoshbakht, T., Hussen, B. M., Taheri, M. N., Ebrahimzadeh, K., & Noroozi, R. (2022) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2022.987174 · Cited by 22 · see citing works
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities
Oikemus, S., Pfister, E. L., Sapp, E., Chase, K. O., Kennington, L. A., Hudgens, E. D., Miller, R., Zhu, L. J., Chaudhary, A., Mick, E. O., et al. (2021) · Human Gene Therapy · doi:10.1089/hum.2020.323 · Cited by 18 · see citing works
Self-citation
Therapeutic targeting of Huntington's disease: Molecular and clinical approaches
Kumar, D., Hasan, G. M., Islam, A., & Hassan, M. I. (2023) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2023.02.075 · Cited by 18 · see citing works
Gene Therapy for Rare Neurological Disorders
Flotte, T. R., & Gessler, D. J. (2022) · Clinical Pharmacology & Therapeutics · doi:10.1002/cpt.2543 · Cited by 20 · see citing works
Modifier pathways in polyglutamine (PolyQ) diseases: from genetic screens to drug targets
Costa, M. D., & Maciel, P. (2022) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-022-04280-8 · Cited by 18 · see citing works
The synaptic lipidome in health and disease
Vallés, A. S., & Barrantes, F. J. (2022) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2022.184033 · Cited by 16 · see citing works
Exploring the Role of Gene Therapy for Neurological Disorders
Puranik, N., Yadav, D., Chauhan, P., Kwak, M., & Jin, J. (2020) · Current Gene Therapy · doi:10.2174/1566523220999200917114101 · Cited by 17 · see citing works
Bioinformatic analysis of microRNA expression in Huntington's disease
Dong, X., & Cong, S. (2018) · Molecular Medicine Reports · doi:10.3892/mmr.2018.9238 · Cited by 16 · see citing works
Emerging pharmacological approaches for Huntington's disease
Singh, K., Jain, D., Sethi, P., Gupta, J. K., Tripathi, A. K., Kumar, S., Sarker, S. D., Nahar, L., & Guru, A. (2024) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2024.176873 · Cited by 14 · see citing works
Gene therapy and immunotherapy as promising strategies to combat Huntington’s disease-associated neurodegeneration: emphasis on recent updates and future perspectives
Jamwal, S., Elsworth, J. D., Rahi, V., & Kumar, P. (2020) · Expert Review of Neurotherapeutics · doi:10.1080/14737175.2020.1801424 · Cited by 12 · see citing works
Emerging strategies for the genetic dissection of gene functions, cell types, and neural circuits in the mammalian brain
Gong, L., Liu, X., Wu, J., & He, M. (2021) · Molecular Psychiatry · doi:10.1038/s41380-021-01292-x · Cited by 8 · see citing works
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
Cabrera, G. T., Meijboom, K. E., Abdallah, A., Tran, H., Foster, Z. W., Weiss, A., Wightman, N., Stock, R. E., Gendron, T. F., Gruntman, A. M., et al. (2023) · Gene Therapy · doi:10.1038/s41434-023-00418-w · Cited by 11 · see citing works
Self-citation
Mutant-Huntingtin Molecular Pathways Elucidate New Targets for Drug Repurposing
Makeeva, V. S., Dyrkheeva, N. S., Lavrik, O. I., Zakian, S. M., & Malakhova, A. A. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms242316798 · Cited by 11 · see citing works
Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration
Mondo, E., Moser, R. P., Gao, G., Mueller, C., Sena‐Esteves, M., Sapp, E., Pfister, E. L., O'Connell, D. C., Takle, K. A., Erger, K. E., et al. (2018) · Journal of Huntington s Disease · doi:10.3233/jhd-180302 · Cited by 10 · see citing works
Self-citation
Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study
Morais, R. D. V. S., Sogorb-González, M., Bar, C., Timmer, N. C., van der Bent, M. L., Wartel, M., & Vallès, A. (2022) · Cells · doi:10.3390/cells11172748 · Cited by 10 · see citing works
Design of AAV Vectors for Delivery of RNAi
Borel, F., & Mueller, C. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_1 · Cited by 10 · see citing works
Self-citation
Revisiting cell and gene therapies in Huntington’s disease
Beatriz, M., Lopes, C., Ribeiro, A. C., & Rego, A. C. (2021) · Journal of Neuroscience Research · doi:10.1002/jnr.24845 · Cited by 10 · see citing works
Attitudes of Potential Participants Towards Molecular Therapy Trials in Huntington’s Disease
Bardakjian, T., Naczi, K. F., & Gonzalez‐Alegre, P. (2019) · Journal of Huntington s Disease · doi:10.3233/jhd-180328 · Cited by 9 · see citing works
Deep brain electrophysiology in freely moving sheep
Perentos, N., Krstulović, M., & Morton, A. J. (2022) · Current Biology · doi:10.1016/j.cub.2021.12.035 · Cited by 7 · see citing works
Self-citation
Adeno-Associated Viral Vectors as Versatile Tools for Neurological Disorders: Focus on Delivery Routes and Therapeutic Perspectives
Fajardo‐Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, Á., Vázquez, A., et al. (2022) · Biomedicines · doi:10.3390/biomedicines10040746 · Cited by 9 · see citing works
The Neuroprotective Properties, Functions, and Roles of Cannabis sativa in Selected Diseases Related to the Nervous System
Mahdi, O., Baharuldin, M. T. H., Nor, N. H. M., Chiroma, S. M., Jagadeesan, S., & Moklas, M. A. M. (2021) · Central Nervous System Agents in Medicinal Chemistry · doi:10.2174/1871524921666210127110028 · Cited by 7 · see citing works
MicroRNAs and Long Non-Coding RNAs Affect the Mechanisms Involved in Age-Related Neurodegeneration in a Manner Depending on RNA-Binding Proteins
Hashemi, M., Asheghabadi, P. S., Moassesfar, M., Mashhadikhan, S., Nasirzade, S., Farahani, A. V., Mehdizadeh, S., Minaei, S., Rahmani, M., Jamshidian, F., et al. (2026) · Molecular Neurobiology · doi:10.1007/s12035-025-05510-3 · Cited by 7 · see citing works
Revolutionizing Huntington’s Disease Treatment: Breakthroughs in AAV-Mediated Gene Therapy
Gavgani, P. M., & García‐Domínguez, M. (2025) · Cells · doi:10.3390/cells14191514 · Cited by 5 · see citing works
An Overview of Gene Editing Modalities and Related Non-clinical Testing Considerations
Vats, S., Ballesteros, C., Hung, S., Sparapani, S., Wong, K., Haruna, J., Li, C., & Authier, S. (2023) · International Journal of Toxicology · doi:10.1177/10915818231153996 · Cited by 4 · see citing works
A one-health approach to using sheep in research, with a focus on neuroscience studies
Chatzimanou, T., & Tsingotjidou, A. (2023) · One Health & Implementation Research · doi:10.20517/ohir.2023.13 · Cited by 4 · see citing works
Neurological Examination of Sheep (Ovis aries) with Unilateral and Bilateral Quinolinic Acid Lesions of the Striatum Assessed by Magnetic Resonance Imaging
O’Connell, A., Sinnott, B., Kuchel, T. R., Perumal, S. R., Fraser, C. K., Hemsley, K. M., & Morton, A. J. (2019) · Journal of Neurology and Experimental Neuroscience · doi:10.17756/jnen.2019-054 · Cited by 4 · see citing works
Self-citation
Imaging Glucose Metabolism and Dopaminergic Dysfunction in Sheep (Ovis aries) Brain Using Positron Emission Tomography Imaging Reveals Abnormalities in OVT73 Huntington’s Disease Sheep
Williams, G. K., Akkermans, J., Lawson, M., Syta, P., Staelens, S. G., Adhikari, M. H., Morton, A. J., Nitzsche, B., Boltze, J., Christou, C., et al. (2024) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.4c00561 · Cited by 3 · see citing works
Self-citation
MicroRNAs in neurodegenerative diseases: from molecular mechanisms to clinical biomarkers, detection methods and therapeutic strategies—advances and challenges
Azam, H. M. H., Mumtaz, M., Rödiger, S., Schierack, P., Hussain, N., & Ambreen, A. (2025) · Neurological Sciences · doi:10.1007/s10072-025-08419-w · Cited by 3 · see citing works
Genselektive Therapieansätze bei der Huntington-Krankheit
Mühlbäck, A., Lindenberg, K. S., Carsten Saft, C., Priller, J., & Landwehrmeyer, G. B. (2020) · Der Nervenarzt · doi:10.1007/s00115-020-00882-4 · Cited by 2 · see citing works
Huntington’s Disease: Les Jeux Sont Faits?
Pfister, E. L., & D, N. A. M. (2020) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2020.08.004 · Cited by 2 · see citing works
Self-citation
Connections between the internal and the external capsules and the globus pallidus in the sheep: A dichromate stain X‐ray microtomographic study
Murillo‐González, J., Notario, B., Maldonado, E., Martínez‐Sanz, E., Asensio, M. D. C. B., & Herrera, M. (2020) · Anatomia Histologia Embryologia · doi:10.1111/ahe.12604 · Cited by 1 · see citing works
Levels of Synaptic Proteins in Brain and Neurofilament Light Chain in Cerebrospinal Fluid and Plasma of OVT73 Huntington’s Disease Sheep Support a Prodromal Disease State
Sapp, E., Boudi, A., Reid, S. J., Trombetta, B. A., Kivisäkk, P., Taghian, T., Arnold, S. E., Howland, D., Gray‐Edwards, H. L., Kegel-Gleason, K. B., et al. (2023) · Journal of Huntington s Disease · doi:10.3233/jhd-230590 · Cited by 1 · see citing works
Self-citation
Huntingtin lowering therapeutics
D, N. A. M., Sena‐Esteves, M., Khvorova, A., DiFiglia, M., & Brodsky, M. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00004-2 · Cited by 1 · see citing works
Self-citation
Huntington's disease: From large animal models to HD gene therapy
Yan, S., Li, X., & Li, S. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00014-5 · Cited by 1 · see citing works
Development of an acute excitotoxic model of Huntington's disease in sheep
O’Connell, A. (2020) · Adelaide Research & Scholarship (AR&S) (University of Adelaide)
Multimodal treatment strategies in Huntington’s disease
Dutta, R. (2021) · Journal of Neuroscience and Neurological Disorders · doi:10.29328/journal.jnnd.1001054
Scaling up to meet new challenges of brain and behaviour through deep brain electrophysiology in freely moving sheepPreprint
Perentos, N., Krstulović, M., & Morton, A. J. (2021) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2021.08.04.454778
Self-citation
Faculty Opinions recommendation of Adeno-associated virus vector as a platform for gene therapy delivery.
Fehlings, M. G. (2020) · Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature · doi:10.3410/f.734958350.793573257
Scaling Up to Meet New Challenges of Brain and Behaviour: Deep Brain Electrophysiology in Freely Moving SheepPreprint
Perentos, N., Krstulović, M., & Morton, A. J. (2021) · SSRN Electronic Journal · doi:10.2139/ssrn.3917173
Self-citation
Protein – protein Interaction Mapping of Neurodegenerative Disease
Samanta, A., & Ghosh, S. (2023) · Journal of Survey in Fisheries Sciences · doi:10.53555/sfs.v10i1s.2163
Neuroengineering: History, modeling, and deliverables
Lucot, K. L., Napoliello, R. E., Vij, P. A., & Fink, K. D. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95730-4.00007-x
Characterising a New Sheep Model of Parkinson's Disease Using Unilateral Intracerebral Injection of 6‐Hydroxydopamine Into the Substantia Nigra
Banstola, A., Vautrelle, N., Bergin, D. H., Mohammad, Y., Gandhi, K., Rizwan, S. B., & Reynolds, J. N. J. (2025) · European Journal of Neuroscience · doi:10.1111/ejn.16668
Advances in Gene Therapy for Neurologic Disorders: An Overview
Chand, P., Koduru, T. S., Chauhan, A. S., Chakraborty, S., & Jakhmola, V. (2025) · BIO Integration · doi:10.15212/bioi-2024-0060
A Deformation Prediction Method with Experimental Validation for 4D Printing ApplicationsPreprint
Bai, J. (2025) · Research Square · doi:10.21203/rs.3.rs-8051468/v1
Developing Interactive Visualisation Systems to Support Design–Engineering Decision-Making in 4D Printing EvaluationPreprint
Bai, J. (2026) · SSRN Electronic Journal · doi:10.2139/ssrn.6616510
Developing Interactive Visualisation Systems to Support Design–Engineering Decision-Making in 4D Printing EvaluationPreprint
Bai, J., Pei, E., Elsouri, M., & perry, M. (2026) · SSRN Electronic Journal · doi:10.2139/ssrn.6836271
A Deformation Estimation Method with Experimental Demonstration for 4D Printing (3D printing and deformation) Design ApplicationsPreprint
Bai, J., Pei, E., & Elsouri, M. (2026) · Research Square · doi:10.21203/rs.3.rs-9410398/v1
Crossing the blood–brain barrier: Advances and frontiers of AAV gene therapy in central nervous system disorders
Han, W., & Zi-Long, Q. I. U. (2026) · Shengming kexue · doi:10.3724/cbls.2026070
Concepts, types, and treatment strategies for neurodegenerative disorders
Rana, R., Jaiswal, S., Sethi, M., Tripathi, S., Mishra, K., Gupta, P., & Chourasia, M. K. (2026) · Elsevier eBooks · doi:10.1016/b978-0-443-33623-2.00003-9
Huntington's and Other Neurodegenerative Disorders
Jain, M., Dhariwal, R., Lahon, A., Bhardwaj, R., & Thakkar, K. (2026) · doi:10.1002/9781394360260.ch10
AAV Vector‐Mediated Modulation of Signaling Pathways in Neurological Disorders: Insights From Cellular, Animal, and Human Studies
Farrokhi, M. R., & Hosseini, K. (2026) · Cell Biochemistry and Function · doi:10.1002/cbf.70270
MicroRNAs in polyglutamine diseases: Mechanistic insights, circulating biomarkers, and emerging microRNA-based therapeutic strategies
Liu, Y., Cui, Y., Sun, M., Gong, L., Liu, K., Zhang, S., Tan, X., & Cong, S. (2026) · Neurobiology of Disease · doi:10.1016/j.nbd.2026.107603
Gene therapy in Huntington’s disease (HD)
Tirgar, F., Hosseini, M., Alijanpour, S., Aghanoori, M.-R., Alijanpour, S., & Mahdavinataj, H. (2026) · Elsevier eBooks · doi:10.1016/b978-0-443-40467-2.00011-7
BindingDB Entry 50018160: The Emerging Landscape of Small-Molecule Therapeutics for the Treatment of Huntington's Disease.
BindingDB (2024) · UC San Diego · doi:10.7270/q28056t7

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease
Caron, N. S., Southwell, A. L., Brouwers, C. C., Cengio, L. D., Xie, Y., Black, H. F., Anderson, L. M., Ko, S., Zhu, X., van Deventer, S. J. H., et al. (2019) · Nucleic Acids Research · doi:10.1093/nar/gkz976 · Cited by 74 · see citing works
Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin
Pfister, E. L., Chase, K. O., Sun, H., Kennington, L. A., Conroy, F., Johnson, E., Miller, R., Borel, F., D, N. A. M., & Mueller, C. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.04.011 · Cited by 30 · see citing works
Self-citation
Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo
Mas, A., Wilson, M. J., Boudreau, R. L., Spengler, R. M., & Davidson, B. L. (2015) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2015.7 · Cited by 38 · see citing works
I02 A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of huntington’s disease
Olejniczak, M., Kotowska‐Zimmer, A., Przybył, Ł., Pewińska, M., Suszyńska-Zajczyk, J., Wronka, D., & Figiel, M. (2021) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2021-ehdn.116
A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease
Kotowska‐Zimmer, A., Przybył, Ł., Pewińska, M., Suszyńska-Zajczyk, J., Wronka, D., Figiel, M., & Olejniczak, M. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.04.031 · Cited by 30 · see citing works

Authors: Pfister, E. L., DiNardo, N., Mondo, E., Borel, F., Conroy, F., Fraser, C., Gernoux, G., Han, X., Hu, D., Johnson, E., Kennington, L., Liu, P., Reid, S. J., Sapp, E., Vodicka, P., Kuchel, T., Morton, A. J., Howland, D., Moser, R., Sena-Esteves, M., Gao, G., Mueller, C., DiFiglia, M., & Aronin, N.
Affiliations: University of Massachusetts Chan Medical School (US)
South Australian Health and Medical Research Institute (AU)
Sichuan University (CN)
Dartmouth College (US)
University of Auckland (NZ)
Massachusetts General Hospital (US)
MassGeneral Institute for Neurodegenerative Disease, Charlestown, Massachusetts.
University of Cambridge (GB)
CHDI Foundation (US)
Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts.
Department of Medicine, University of Massachusetts Medical School, Worcester, Massachusetts.
ORCID: Pfister, Edith L.
Mondo, Erica
Borel, Florie
Conroy, Faith
Gernoux, Gwladys
Han, Xin
Johnson, Emily
Liu, PengPeng
Reid, Suzanne J.
Sapp, Ellen
Kuchel, Tim
Morton, A. Jennifer
Howland, David
Moser, Richard
Gao, Guangping
Mueller, Christian
DiFiglia, Marian
Venue: Human Gene Therapy
Publisher: SAGE Publications
Published: 2018-06
Volume: 29
Issue: 6
Pages: 663-673
ISSN: 1043-0342 (ISSN-L)
1557-7422
DOI: 10.1089/hum.2017.199
PMC: PMC6909722
OpenAlex: W2774783374
Cited by: 95
References: 30
Citations by year: 2026: 10 · 2025: 5 · 2024: 5 · 2023: 9 · 2022: 15 · 2021: 16 · 2020: 14 · 2019: 14 · 2018: 6 · 2016: 1
FWCI: 4.55
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.liebertpub.com/nv/resources-tools/text-and-data-mining-policy/121/ (tdm)
Locations: Human Gene Therapy (publishedVersion)
Europe PMC (PubMed Central) (publishedVersion)
PubMed Central (publishedVersion)
PubMed (publishedVersion)
eScholarship@UMassChan (submittedVersion)
Apollo (University of Cambridge) (submittedVersion)
Apollo
Funders: National Institute of Neurological Disorders and Stroke (R01NS088689)
Topics: Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Keywords: Huntington's disease, artificial microRNA, mutant huntingtin, gene silencing, striatum, CAG repeat expansion, alternative promoters, neuroprotection, microglia, large animal model
MeSH terms: Huntingtin Protein, Dependovirus, Animals, Disease Models, Animal, Electrolytes, Genetic Vectors, Humans, Huntington Disease, Immunoassay, Injections, Kidney, Liver, Neurons, RNA, Messenger, Sheep, Genome, Viral, Neostriatum, Microglia, Animals, Genetically Modified, MicroRNAs, Mutant Proteins

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Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

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