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MicroRNAs regulating autophagy: opportunities in treating neurodegenerative diseases

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Pathobiology of the autophagy-lysosomal pathway in the Huntington’s disease brainPreprint

Berg, M. J., Veeranna, Rosa, C. M., Kumar, A. R., Mohan, P. S., Stavrides, P. H., Marchionini, D. M., Yang, D., & Nixon, R. A. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.05.29.596470 · Cited by 12 · see citing works

Gastrodin Improves the Activity of the Ubiquitin–Proteasome System and the Autophagy–Lysosome Pathway to Degrade Mutant Huntingtin

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Small molecule modulation of p75NTR engages the autophagy-lysosomal pathway and reduces huntingtin aggregates in cellular and mouse models of Huntington's disease

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Fate erasure logic of gene networks underlying direct neuronal conversion of somatic cells by microRNAs

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Screens in aging-relevant human ALS-motor neurons identify MAP4Ks as therapeutic targets for the disease

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REM Sleep Loss-Induced Elevated Noradrenaline Plays a Significant Role in Neurodegeneration: Synthesis of Findings to Propose a Possible Mechanism of Action from Molecule to Patho-Physiological Changes

Giri, S., Mehta, R., & Mallick, B. N. (2023) · Brain Sciences · doi:10.3390/brainsci14010008 · Cited by 9 · see citing works

A pathogenic Tau mutation drives autophagy-lysosome dysfunction that limits Tau degradation in a model of frontotemporal dementia

Mirfakhar, F. S., Marsh, J. A., Sato, C., Schache, K. J., Minaya, M., Dolle, R. E., Pak, S. C., Silverman, G. A., Perlmutter, D. H., Macauley, S. L., et al. (2026) · Nature Communications · doi:10.1038/s41467-026-70473-5 · Cited by 9 · see citing works

Age‐ and disease‐related autophagy impairment in Huntington disease: New insights from direct neuronal reprogramming

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Pathogenesis of polyglutamine diseases: Piecing together a complex molecular puzzle

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Autophagy–NAD+ axis: emerging insights into neuronal homeostasis and neurodegenerative diseases

Koçak, G., Dimas, M. M., Silva, L., Silva-Amaral, D., Sun, C., Cruddas, S., Barrett, T. G., Martins‐de‐Souza, D., Cunha‐Neto, E., Brocardo, P. S., et al. (2025) · Frontiers in Molecular Biosciences · doi:10.3389/fmolb.2025.1695486 · Cited by 8 · see citing works

Patient-derived neuron model: Capturing age-dependent adult-onset degenerative pathology in Huntington’s disease

Oh, Y. M., & Lee, S. W. (2024) · Molecules and Cells · doi:10.1016/j.mocell.2024.100046 · Cited by 7 · see citing works

Deciphering disease signatures and molecular targets in vascular Ehlers-Danlos syndrome through transcriptome and miRNome sequencing of dermal fibroblasts

Chiarelli, N., Cinquina, V., Martini, P. G. V., Bertini, V., Zoppi, N., Venturini, M., Ritelli, M., & Colombi, M. (2023) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2023.166915 · Cited by 7 · see citing works

Multiple Genes Core to ERAD, Macroautophagy and Lysosomal Degradation Pathways Participate in the Proteostasis Response in α1-Antitrypsin Deficiency

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Advancing Ischemic Stroke Prognosis: Key Role of MiR-155 Non-Coding RNA

Hering, C., & Conover, G. M. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26093947 · Cited by 6 · see citing works

Absence of Rab39b-induced macroautophagy impairment increases neurotoxic α-synuclein and causes degeneration of substantia nigra dopaminergic neurons in mouse model of X-linked Parkinson's disease

Chiu, C., Weng, Y.-H., Yeh, T., Chen, W.-S., Liu, S.-Y., Chiu, T.-J., Li, A. H.-L., & Wang, H. (2025) · Life Sciences · doi:10.1016/j.lfs.2025.123789 · Cited by 6 · see citing works

Insights and applications of direct neuronal reprogramming

Schaukowitch, K., Janas, J. A., & Wernig, M. (2023) · Current Opinion in Genetics & Development · doi:10.1016/j.gde.2023.102128 · Cited by 7 · see citing works

Inborn errors of canonical autophagy in neurodegenerative diseases

Freisem, D., Hoenigsperger, H., Catanese, A., & Sparrer, K. M. J. (2024) · Human Molecular Genetics · doi:10.1093/hmg/ddae179 · Cited by 6 · see citing works

Comparison of Oncogenes, Tumor Suppressors, and MicroRNAs Between Schizophrenia and Glioma: The Balance of Power

Brown, J. S. (2023) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2023.105206 · Cited by 6 · see citing works

Reversing memory/cognitive impairment with medicinal plants targeting inflammation and its crosstalk with other pathologies

Tyler, S. E. B., & Tyler, L. D. K. (2023) · Brain Disorders · doi:10.1016/j.dscb.2023.100094 · Cited by 6 · see citing works

Microbiota dysbiosis impact on the immune system dysregulation in Huntington’s disease (HD)

Acharjee, P., Prasad, S. K., Singh, V. V., Ray, M., & Acharjee, A. (2025) · International review of neurobiology · doi:10.1016/bs.irn.2025.04.002 · Cited by 5 · see citing works

Moving CNS axon growth and regeneration research into human model systems

Lear, B. P., & Moore, D. L. (2023) · Frontiers in Neuroscience · doi:10.3389/fnins.2023.1198041 · Cited by 5 · see citing works

Dedifferentiation-like reprogramming of degenerative nucleus pulposus cells into notochordal-like cells by defined factors

Zhang, Y., Liang, C., Xu, H., Li, Y., Xia, K., Wang, L., Huang, X., Chen, J., Shu, J., Cheng, F., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.06.018 · Cited by 6 · see citing works

Pathobiology of the autophagy-lysosomal pathway in the Huntington’s disease brain

Berg, M. J., Veeranna, Rosa, C. M., Kumar, A. R., Mohan, P. S., Stavrides, P. H., Darji, S., Marchionini, D. M., Yang, D., & Nixon, R. A. (2025) · Acta Neuropathologica Communications · doi:10.1186/s40478-025-02131-8 · Cited by 15 · see citing works

Navigating the Autophagy Maze: ATG and Their Impact on Neurodegenerative Diseases

Amirian, R., Merati, A., Babamohamadi, M., Mirahmadi, Y., Esfahani, M. L., Rahmani, S., Izadi, Z., & Rezazadeh, D. (2025) · Molecular Neurobiology · doi:10.1007/s12035-025-05332-3 · Cited by 4 · see citing works

Fountain of youth—Targeting autophagy in aging

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Programming human cell fate: overcoming challenges and unlocking potential through technological breakthroughs

Lin, H., Makhlouf, A., Echegaray, C. V., Zawada, D. M., & Simões, F. C. (2023) · Development · doi:10.1242/dev.202300 · Cited by 4 · see citing works

Direct Reprogramming of Somatic Skin Cells from a Patient with Huntington’s Disease into Striatal Neurons to Create Models of Pathology

Kraskovskaya, N., Георгиевич, Х. М., Tomilin, A. N., & Mikhailova, N. (2024) · Doklady Biological Sciences · doi:10.1134/s0012496623700849 · Cited by 4 · see citing works

Multi-OMIC analysis of Huntington disease reveals a neuroprotective astrocyte statePreprint

Paryani, F., Kwon, J., Ng, C. W., Madden, N., Ofori, K., Tang, A., Lü, H., Li, J., Mahajan, A., Davidson, S. M., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.09.08.556867 · Cited by 4 · see citing works

Efficacy of therapy by MK-28 PERK activation in the Huntington's disease R6/2 mouse model

Shacham, T., Offen, D., & Lederkremer, G. Z. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00335 · Cited by 3 · see citing works

Inhibition of UBE2N promotes the clearance of mutant HTT (huntingtin) in HD knock-in mice

Ou, K., Wang, X., Guo, M., Li, D., Zhang, C., Chen, L., Hou, J., Jia, Q., Zhu, L., Yang, S., et al. (2025) · Autophagy · doi:10.1080/15548627.2025.2549109 · Cited by 3 · see citing works

Short Peptides Protect Fibroblast-Derived Induced Neurons from Age-Related Changes

Kraskovskaya, N., Linkova, N. S., Sakhenberg, E. I., Kriger, D. V., Polyakova, V. O., Медведев, Д. С., Krasichkov, A. S., Георгиевич, Х. М., & Ryzhak, G. A. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms252111363 · Cited by 3 · see citing works

Metabolism-Mediated FGF5 Association with Stroke: Based on Mendelian Randomization and Bioinformatics Analysis

Xu, C., Xu, Y., Gao, L., Wang, M., Wang, G., & Wang, G. (2025) · Journal of Multidisciplinary Healthcare · doi:10.2147/jmdh.s529168 · Cited by 2 · see citing works

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

Targeting UCHL3 attenuates pathological markers in neuronal models of Huntington’s disease

Ishtayeh, H., Battistoni, E., Pochtar, S., McHugh, T. L. M., Tshilenge, K., Rossmiller, B. P., Amer‐Sarsour, F., Berdichevsky, Y., Muchtar, N., Weil, M., et al. (2026) · Brain · doi:10.1093/brain/awag028 · Cited by 2 · see citing works

SerpinA3N inhibits mitochondrial complex I activity to prevent neuron ferroptosis following cerebral ischemic stroke

Liu, X., Li, G., Guo, Y., Li, R., Yi, S., Ding, S., & Wan, J. (2025) · Experimental Neurology · doi:10.1016/j.expneurol.2025.115370 · Cited by 2 · see citing works

Autophagy and Huntington's disease

Croce, K. R., Jasutkar, H. G., & Yamamoto, A. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00018-2 · Cited by 2 · see citing works

Chemical screens in aging-relevant human motor neurons identify MAP4Ks as therapeutic targets for amyotrophic lateral sclerosisPreprint

Liu, M., Ma, S., Tai, W., Zhong, X., Ni, H., Zou, Y., wang, J., & Zhang, C. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.04.24.538014 · Cited by 2 · see citing works

Robust CRISPR/dCas13 RNA blockers specifically perturb miRNA-target interactions and rescue type 1 myotonic dystrophy pathologyPreprint

Hanifi, M., Ateş, P. S., Wen, S., Fischer, M., Kroesen, A. E., Yu, Z., Wood, M., Thongjuea, S., Mead, A. J., Fulga, T. A., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.09.16.612263 · Cited by 2 · see citing works

Striatal neuronal models of Huntington's disease via direct conversion: Modeling age-dependent disease phenotypes

Oh, Y. M., Lee, S. W., & Yoo, A. S. J. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00012-1 · Cited by 1 · see citing works

Autophagy-mediated cell death: Mechanisms and implications in neurodegenerative disorders

Zhang, L., Lü, Y., & Liu, B. (2025) · Elsevier eBooks · doi:10.1016/b978-0-443-32852-7.00017-6 · Cited by 1 · see citing works

Genetic dissection of Huntington’s disease modification by variation at RRM2B

Lee, K., Shin, B., Kim, M., Lee, S. W., Oh, Y. M., Kim, K., Jiang, A., Ko, K., Gillis, T., Lucente, D. E., et al. (2025) · Human Molecular Genetics · doi:10.1093/hmg/ddaf150 · Cited by 1 · see citing works

Hydrogen Sulfide Signaling in Neurodegenerative Movement Disorders

Pieper, A. A., & Paul, B. D. (2025) · Handbook of experimental pharmacology · doi:10.1007/164_2025_757 · Cited by 1 · see citing works

Targeting STAT3 in Alzheimer's disease: Potential mechanisms and therapeutic implications.

Chen, P., Wang, R., Zhou, T., Sun, Y., Kirchhoff, F., Miao, Z., & Zhuang, C.-L. (2026) · European journal of medicinal chemistry · doi:10.1016/j.ejmech.2026.118785 · Cited by 1 · see citing works

DHCR24 in cholesterol metabolism and diseases of the nervous system

Zhu, H. G., Li, S., Yang, Y. Z., 张似青, Wang, Y., & Xiong, T. (2026) · Lipids in Health and Disease · doi:10.1186/s12944-026-02954-x · Cited by 1 · see citing works

Three-dimensional direct neuronal reprogramming for modeling Alzheimer’s disease neuropathology

Sun, Z., Lee, Y., Walker, C. K., Karch, C. M. M., & Yoo, A. S. J. (2026) · Nature Protocols · doi:10.1038/s41596-026-01388-0 · Cited by 1 · see citing works

Roles of autophagy in brain homeostasis and disease

Kim, H., Wickstead, E. S., Zhou, X., Choi, I., & Yue, Z. (2026) · Nature Neuroscience · doi:10.1038/s41593-026-02426-6 · Cited by 1 · see citing works

Advancements in personalized stem cell models for aging-related neurodegenerative disorders

Weng, M., & Jauch, R. (2024) · Neural Regeneration Research · doi:10.4103/nrr.nrr-d-23-01793 · Cited by 1 · see citing works

Suppression of the lncRNA TUG1 alleviates neuropathic pain in rats with chronic contractile injury via the miR-29b-3p/HMGB1 axis

Dong, J., Ding, Y., Xia, G., Guo, X., Jiang, L., & Ouyang, A. (2024) · Folia Neuropathologica · doi:10.5114/fn.2024.141512 · Cited by 1 · see citing works

Dermal Fibroblast Cell Line from a Patient with the Huntington’s Disease as a Promising Model for Studying Disease Pathogenesis: Production and Characterization

Kraskovskaya, N., Koltsova, A. M., Parfenova, P. S., Shatrova, A. N., Yartseva, N., Назаров, В. Д., Devyatkina, E., Георгиевич, Х. М., & Mikhailova, N. (2024) · Biochemistry (Moscow) · doi:10.1134/s000629792407006x · Cited by 1 · see citing works

Diagnostic Value of Serum miR-499a-5p in Chinese Children with Autism Spectrum Disorders

Ren, J., Bai, Y., Gao, J., Hou, Y., Mao, J., Gao, F., & Wang, J. (2025) · Journal of Molecular Neuroscience · doi:10.1007/s12031-024-02296-2 · Cited by 1 · see citing works

Age-maintained human neurons demonstrate a developmental loss of intrinsic neurite growth abilityPreprint

Lear, B. P., Thompson, E. A. N., Rodriguez, K., Arndt, Z. P., Khullar, S., Klosa, P. C., Lu, R. J., Morrow, C. S., Risgaard, R. D., Peterson, E. R., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.05.23.541995 · Cited by 1 · see citing works

SAK3 confers neuroprotection in the neurodegeneration model of X-linked Dystonia-ParkinsonismPreprint

Aryal, S., Chen, S., Burbach, K. F., Yang, Y., Capano, L. S., Kim, W. K., Bragg, D. C., & Yoo, A. S. J. (2024) · Research Square · doi:10.21203/rs.3.rs-4068432/v1 · Cited by 1 · see citing works

Autophagic enhancer rescues Tau accumulation in a stem cell model of frontotemporal dementiaPreprint

Mirfakhar, F. S., Marsh, J. A., Minaya, M., Pak, S. C., Silverman, G. A., Perlmutter, D. H., Macauley, S. L., & Karch, C. M. M. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.09.30.615921 · Cited by 1 · see citing works

Sequelae and reversal of age-dependent alterations in mitochondrial dynamics via autophagy enhancement in reprogrammed human neuronsPreprint

Klinman, E., Kwon, J., Dolle, R. E., Pak, S. C., Silverman, G. A., Perlmutter, D. H., & Yoo, A. S. J. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.02.680077 · Cited by 1 · see citing works

Neuronal aging is associated with declined autophagy caused by reduced functionality of cGAS-STING signalingPreprint

Passarella, S., Brandes, K., Dankert, E., Cavalli, P., Kröger, A., Dieterich, D. C., & Landgraf, P. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.09.25.559304

Age-related disease: Central nervous system

Fossel, M. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-15500-0.00005-0

Age-dependent alterations in the human striatum identify a PPP1R15B-miR-196a node as a modifier of Huntington’s disease phenotypesPreprint

Yoo, A. S. J., Church, V. A., Krzyżosiak, A., Chen, S., Kwon, J., Victor, M. B., Miao, B., Zhang, B., Dahiya, S. M., & Bertolotti, A. (2024) · Research Square · doi:10.21203/rs.3.rs-4104643/v1

Direct reprogramming of somatic skin cells from a patient with Huntington's disease into striatal neurons to create models of pathology

Kraskovskaya, N., Георгиевич, Х. М., Tomilin, A. N., & Mikhailova, N. (2024) · Доклады Российской академии наук Науки о жизни · doi:10.31857/s2686738924010131

Dermal Fibroblast Lines from a Patient with Huntington’s Disease as a Promising Model for Studying the Pathogenesis of the Disease: Production and Characterization

Kraskovskaya, N., Koltsova, A. M., Parfenova, P. S., Shatrova, A. N., Yartseva, N., Назаров, В. Д., Devyatkina, E., Георгиевич, Х. М., & Mikhailova, N. (2024) · Biochemistry (Moscow) · doi:10.31857/s0320972524070046

spRefine Denoises and Imputes Spatial Transcriptomics with a Reference-Free Framework Powered by Genomic Language ModelPreprint

Liu, T., Huang, T., Jin, W., Chu, T., Ying, R., & Zhao, H. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.04.22.649977

Post-transcriptional regulation in intervertebral disc degeneration

Li, X., Liang, H., Du, Z., Shi, R., Song, Y., & Yang, C. (2026) · Genes & Diseases · doi:10.1016/j.gendis.2026.102037

Decoding Non-Neuronal Mechanisms and Therapeutic Targets in Huntington’s Disease Through Integrative Transcriptomics and Machine Learning

Gupta, H., Singh, S., Kaushik, A. C., Awasthi, A. K., & Sahi, S. (2026) · Journal of Molecular Neuroscience · doi:10.1007/s12031-025-02463-z

Large-scale Perturbation of Systems Biology-Derived Genes Reveals Modifiers of HD- associated Transcriptomic Networks and PathologyPreprint

Langfelder, P., Wang, N., Ramanathan, L., Oh, Y. M., Lee, S., Gao, F., Gu, X., Stricos, M., Plascencia, M., Vaca, R., et al. (2026) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2026.03.02.709091

Deubiquitinases at organelle quality control bottlenecks in neurodegeneration

Ellerby, L., & Ashkenazi, A. (2026) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2026.05.006

CDKN1A protects medium spiny neurons from Huntington's disease pathology

Lee, S. W., Upshaw, T. J., Bailey, D. J., Lee, S.-A., Kim, J., Joshi, P., Smith, G. E., Swinford, S., & Oh, Y. M. (2026) · Neurobiology of Disease · doi:10.1016/j.nbd.2026.107522

Direct neuronal reprogramming for neurological diseases: applications, translational Challenges, and future directions

Chen, Y., Zheng, Z., Zhao, P., Zhu, J., Sun, H., Zhu, H., Zeng, Y., Shang, B., Zhang, A., Pan, Y., et al. (2026) · Frontiers in Neuroscience · doi:10.3389/fnins.2026.1889859

Sequalae and reversal of age-dependent alterations in mitochondrial dynamics via autophagy enhancement in reprogrammed human neurons

Klinman, E., Kwon, J.-S., Dolle, R. E., Pak, S. C., Silverman, G. A., Perlmutter, D. H., & Yoo, A. S. J. (2026) · Autophagy · doi:10.1080/15548627.2026.2719435 · Cited by 1 · see citing works

Regenerative medicine for neurodegenerative diseases: history, strategies, and clinical advances

Li, J., Duan, H., Hao, P., Zhao, W., Gao, Y., Yang, Z., & Li, X. (2026) · Translational research · doi:10.1016/j.trsl.2026.08.005

Identification of Age-Associated Upstream Regulators to Promote Neuronal Resilience in HD Patient-Derived Neurons

Oh, Y. M., Bacallao, E. A., Lee, S.-A., Kim, J., Swinford, S., Smith, G. E., Britt, B., Abdalla, S. M., & Lee, S. W. (2026) · Neuroscience Bulletin · doi:10.1007/s12264-026-01711-5

miR-196a reduces mutant Huntingtin aggregates by Rad23b-mediated degradation in Huntington’s disease

Tung, C.-W., Chan, S. C., Chen, Y.-C., Wu, P., Cheng, P., Chen, C., & Yang, S. (2026) · Journal of Biomedical Science · doi:10.1186/s12929-026-01292-5

Modeling human neurodegeneration with iPSCs: opportunities, limitations, and the challenge of aging

Wang, S., Elia, L., Liu, W., & Finkbeiner, S. M. (2026) · Journal of Clinical Investigation · doi:10.1172/jci209602

Neuroprotective Role of Withaferin A in Huntington’s Disease: Current Evidence and Future Perspectives

Kumari, R., Gupta, V., Devi, K., Chand, D., Dhanasekaran, M., & Agarwal, S. (2026) · Molecular Neurobiology · doi:10.1007/s12035-026-06274-0

HAP40 functions as a proteostasis regulator by controlling huntingtin interactions and its release into the extracellular space

Ramos, E. S., Boeddrich, A., Haenig, C., Trepte, P., Ammar, O., Secker, C., Popp, O., Ivanov, A., Beetz, S., Roth, L., et al. (2026) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2611301123

Modeling Huntington disease through microRNA-mediated neuronal reprogramming identifies age-associated autophagy dysfunction driving the onset of neurodegeneration

Oh, Y. M., Lee, S. W., & Yoo, A. S. J. (2023) · Autophagy · doi:10.1080/15548627.2023.2175572 · Cited by 20 · see citing works

Striatal neuronal models of Huntington's disease via direct conversion: Modeling age-dependent disease phenotypes

Oh, Y. M., Lee, S. W., & Yoo, A. S. J. (2024) · Elsevier eBooks · doi:10.1016/b978-0-323-95672-7.00012-1 · Cited by 1 · see citing works

Age‐ and disease‐related autophagy impairment in Huntington disease: New insights from direct neuronal reprogramming

Luo, C., & Yang, J. (2024) · Aging Cell · doi:10.1111/acel.14285 · Cited by 9 · see citing works

Patient-derived neuron model: Capturing age-dependent adult-onset degenerative pathology in Huntington’s disease

Oh, Y. M., & Lee, S. W. (2024) · Molecules and Cells · doi:10.1016/j.mocell.2024.100046 · Cited by 7 · see citing works

Identification of Age-Associated Upstream Regulators to Promote Neuronal Resilience in HD Patient-Derived Neurons

Oh, Y. M., Bacallao, E. A., Lee, S.-A., Kim, J., Swinford, S., Smith, G. E., Britt, B., Abdalla, S. M., & Lee, S. W. (2026) · Neuroscience Bulletin · doi:10.1007/s12264-026-01711-5