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Identification and characterization of a spinal muscular atrophy-determining gene

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A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

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Correlation between severity and SMN protein level in spinal muscular atrophy

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Spinal muscular atrophy

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Quantitative Analyses of SMN1 and SMN2 Based on Real-Time LightCycler PCR: Fast and Highly Reliable Carrier Testing and Prediction of Severity of Spinal Muscular Atrophy

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A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2

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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

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The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn-/- mice and results in a mouse with spinal muscular atrophy

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Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis

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Antisense Oligonucleotides (ASOs) in Motor Neuron Diseases: A Road to Cure in Light and Shade

Cantara, S., Simoncelli, G., & Ricci, C. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25094809 · Cited by 44

Dynamic dual-isotope molecular imaging elucidates principles for optimizing intrathecal drug delivery

Wolf, D. A., Hesterman, J. Y., Sullivan, J. M., Orcutt, K. D., Silva, M. D., Lobo, M. R., Wellman, T. J., Hoppin, J., & Verma, A. (2016) · JCI Insight · doi:10.1172/jci.insight.85311 · Cited by 42

Targeted Delivery of Antisense Oligonucleotides Using Neurotensin Peptides

Nikan, M., Tanowitz, M., Dwyer, C. A., Jackson, M., Gaus, H. J., Swayze, E. E., Rigo, F., Seth, P. P., & Prakash, T. P. (2020) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.0c00840 · Cited by 42

Clinical Pharmacokinetics of Approved RNA Therapeutics

Jo, S. J., Chae, S. U., Lee, C. B., & Bae, S. K. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24010746 · Cited by 41

Spinal Muscular Atrophy: Journeying From Bench to Bedside

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Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3

Toonen, L. J. A., Schmidt, I., Luijsterburg, M. S., van Attikum, H., & van Roon‐Mom, W. (2016) · Scientific Reports · doi:10.1038/srep35200 · Cited by 42

Translating Antisense Technology into a Treatment for Huntington’s Disease

Lane, R. M., Smith, A. V., Baumann, T. L., Gleichmann, M., Norris, D., Bennett, C. F., & Kordasiewicz, H. B. (2018) · Methods in molecular biology · doi:10.1007/978-1-4939-7825-0_23 · Cited by 39

Intracerebroventricular Administration of a 2′-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain

Toonen, L. J. A., Casaca-Carreira, J., Pellisé-Tintoré, M., Mei, H., Temel, Y., Jahanshahi, A., & van Roon‐Mom, W. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0705 · Cited by 38

Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction

Wang, L., Kempton, J. B., Jiang, H., Jodelka, F. M., Brigande, A. M., Dumont, R. A., Rigo, F., Lentz, J. J., Hastings, M. L., & Brigande, J. V. (2020) · Nucleic Acids Research · doi:10.1093/nar/gkaa194 · Cited by 44

Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins

Pardridge, W. M. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114234 · Cited by 40

Rescue of gene-expression changes in an induced mouse model of spinal muscular atrophy by an antisense oligonucleotide that promotes inclusion of SMN2 exon 7

Staropoli, J. F., Huo, L., Chun, S. J., Allaire, N., Cullen, P. F., Thai, A., Fleet, C., Hua, Y., Bennett, C. F., Krainer, A. R., et al. (2015) · Genomics · doi:10.1016/j.ygeno.2015.01.007 · Cited by 36

Splicing Regulation: A Molecular Device to Enhance Cancer Cell Adaptation

Pagliarini, V., Naro, C., & Sette, C. (2015) · BioMed Research International · doi:10.1155/2015/543067 · Cited by 36

Antisense oligonucleotides delivered to the amniotic cavityin uteromodulate gene expression in the postnatal mouse

Depreux, F. F. S., Wang, L., Jiang, H., Jodelka, F. M., Rosencrans, R. F., Rigo, F., Lentz, J. J., Brigande, J. V., & Hastings, M. L. (2016) · Nucleic Acids Research · doi:10.1093/nar/gkw867 · Cited by 35

Fatigue in adults with spinal muscular atrophy under treatment with nusinersen

Kizina, K., Stolte, B., Totzeck, A., Bolz, S., Schlag, M., Ose, C., von Velsen, O., Kleinschnitz, C., & Hagenacker, T. (2020) · Scientific Reports · doi:10.1038/s41598-020-68051-w · Cited by 38

NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice

Torres‐Benito, L., Schneider, S. M., Rombo, R., Ling, K., Grysko, V., Upadhyay, A., Kononenko, N. L., Rigo, F., Bennett, C. F., & Wirth, B. (2019) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2019.05.008 · Cited by 42

Antisense antibacterial compounds

Pifer, R., & Greenberg, D. E. (2020) · Translational research · doi:10.1016/j.trsl.2020.06.001 · Cited by 34

In Search of a Cure: The Development of Therapeutics to Alter the Progression of Spinal Muscular Atrophy

Ojala, K. S., Reedich, E. J., DiDonato, C. J., & Meriney, S. D. (2021) · Brain Sciences · doi:10.3390/brainsci11020194 · Cited by 37

Viral Vector-Mediated Antisense Therapy for Genetic Diseases

Imbert, M., Dias-Florencio, G., & Goyenvalle, A. (2017) · Genes · doi:10.3390/genes8020051 · Cited by 35

Neurocalcin Delta Knockout Impairs Adult Neurogenesis Whereas Half Reduction Is Not Pathological

Upadhyay, A., Hosseinibarkooie, S., Schneider, S. M., Kaczmarek, A., Torres‐Benito, L., Mendoza-Ferreira, N., Overhoff, M., Rombo, R., Grysko, V., Kye, M. J., et al. (2019) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2019.00019 · Cited by 33

Therapeutic antisense oligonucleotides for movement disorders

Doxakis, E. (2020) · Medicinal Research Reviews · doi:10.1002/med.21706 · Cited by 34

Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations

Aartsma-Rus, A. M., van Roon‐Mom, W., Lauffer, M. C., Siezen, C. L. E., Duijndam, B., Roo, T. C.-D., Schüle, R., Synofzik, M., & Graeßner, H. (2023) · RNA · doi:10.1261/rna.079540.122 · Cited by 32

The expanding application of antisense oligonucleotides to neurodegenerative diseases

Sumner, C. J., & Miller, T. M. (2024) · Journal of Clinical Investigation · doi:10.1172/jci186116 · Cited by 32

Review of Spinal Muscular Atrophy (SMA) for Prenatal and Pediatric Genetic Counselors

Carré, A., & Empey, C. (2015) · Journal of Genetic Counseling · doi:10.1007/s10897-015-9859-z · Cited by 31

Knockdown of polypyrimidine tract binding protein facilitates motor function recovery after spinal cord injury

Yang, R., Chai, R., Pan, J.-Y., Pan, J., Xia, P.-H., Wang, Y.-K., Chen, Y., Li, Y., Wu, J., & Chen, G. (2022) · Neural Regeneration Research · doi:10.4103/1673-5374.346463 · Cited by 30

The MOE Modification of RNA: Origins and Widescale Impact on the Oligonucleotide Therapeutics Field

Hill, A. C., & Hall, J. (2023) · Helvetica Chimica Acta · doi:10.1002/hlca.202200169 · Cited by 31

Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain

Walters, B. J., Azam, A. B., Gillon, C. J., Josselyn, S. A., & Zovkic, I. B. (2016) · Frontiers in Genetics · doi:10.3389/fgene.2015.00362 · Cited by 31

Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice

Saoudi, A., Barberat, S., Coz, O. L., Vacca, O., Doisy, M., Tensorer, T., Śliwiński, É., Garcı́a, L., Francesco Muntoni, A., Vaillend, C., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.03.009 · Cited by 30

Bio–Nano Toolbox for Precision Alzheimer's Disease Gene Therapy

Liu, Y., Xia, X., Zheng, M., & Shi, B. (2024) · Advanced Materials · doi:10.1002/adma.202314354 · Cited by 31

Antisense Oligonucleotide in LNA-Gapmer Design Targeting TGFBR2—A Key Single Gene Target for Safe and Effective Inhibition of TGFβ Signaling

Kuespert, S., Heydn, R., Peters, S., Wirkert, E., Meyer, A.-L., Siebörger, M., Johannesen, S. W., Aigner, L., Bogdahn, U., & Bruun, T. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21061952 · Cited by 29

Lowering Mutant Huntingtin Using Tricyclo-DNA Antisense Oligonucleotides As a Therapeutic Approach for Huntington's Disease

Imbert, M., Blandel, F., Leumann, C. J., Garcı́a, L., & Goyenvalle, A. (2019) · Nucleic Acid Therapeutics · doi:10.1089/nat.2018.0775 · Cited by 29

Antisense-mediated reduction of EphA4 in the adult CNS does not improve the function of mice with amyotrophic lateral sclerosis

Ling, K., Jackson, M., Alkam, D., Liu, D., Allaire, N., Sun, C., Kiaei, M., McCampbell, A., & Rigo, F. (2018) · Neurobiology of Disease · doi:10.1016/j.nbd.2018.03.002 · Cited by 25

Corticosteroid sensitization drives opioid addiction

Carmack, S. A., Vendruscolo, J. C. M., McGinn, M. A., Miranda-Barrientos, J. A., Repunte‐Canonigo, V., Bossé, G. D., Mercatelli, D., Giorgi, F. M., Fu, Y., Hinrich, A. J., et al. (2022) · Molecular Psychiatry · doi:10.1038/s41380-022-01501-1 · Cited by 29

Intracerebroventricular administration for delivery of antiseizure therapeutics: Challenges and opportunities

Fahoum, F., & Eyal, S. (2023) · Epilepsia · doi:10.1111/epi.17625 · Cited by 27

Progress and promise of antisense oligonucleotide therapeutics for central nervous system diseases

Bishop, K. M. (2016) · Neuropharmacology · doi:10.1016/j.neuropharm.2016.12.015 · Cited by 28

Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

Hu, N., Antoury, L., Baran, T. M., Mitra, S., Bennett, C. F., Rigo, F., Foster, T. H., & Wheeler, T. M. (2018) · Nature Communications · doi:10.1038/s41467-018-07517-y · Cited by 27

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.152203 · Cited by 27

Principles and correction of 5’-splice site selection

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Non-invasive Imaging of Antisense Oligonucleotides in the Brain via In Vivo Click Chemistry

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Recent Progress in Gene-Targeting Therapies for Spinal Muscular Atrophy: Promises and Challenges

Haque, U. S., & Yokota, T. (2024) · Genes · doi:10.3390/genes15080999 · Cited by 27

Advances in modeling and treating spinal muscular atrophy

Alstyne, M. V., & Pellizzoni, L. (2016) · Current Opinion in Neurology · doi:10.1097/wco.0000000000000368 · Cited by 25

Modulation of aberrant splicing in human RNA diseases by chemical compounds

Kataoka, N. (2017) · Human Genetics · doi:10.1007/s00439-017-1789-4 · Cited by 25

Preclinical Safety Assessment of Therapeutic Oligonucleotides

Andersson, P. U. (2022) · Methods in molecular biology · doi:10.1007/978-1-0716-2010-6_25 · Cited by 28

MRI characterization of early CNS transport kinetics post intrathecal gadolinium injection: Trends of subarachnoid and parenchymal distribution in healthy volunteers

Dyke, J. P., Xu, H. S., Verma, A., Voss, H. U., & Chazen, J. L. (2020) · Clinical Imaging · doi:10.1016/j.clinimag.2020.04.043 · Cited by 28

Pharmacomodulation of microRNA Expression in Neurocognitive Diseases: Obstacles and Future Opportunities

Simion, V., Nadim, W. D., Bénédetti, H., Pichon, C., Morisset‐Lopez, S., & Baril, P. (2016) · Current Neuropharmacology · doi:10.2174/1570159x14666160630210422 · Cited by 26

MicroRNAs in brain cholesterol metabolism and their implications for Alzheimer's disease

Yoon, H., Flores, L. F., & Kim, J. (2016) · Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of Lipids · doi:10.1016/j.bbalip.2016.04.020 · Cited by 23

Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy

Borges, B., Brown, S. M., Chen, W., Clarke, M. T., Herzeg, A., Park, J. H., Ross, J., Kong, L., Denton, M., Smith, A. K., et al. (2025) · Science Translational Medicine · doi:10.1126/scitranslmed.adv4656 · Cited by 24

Translational development of splice-modifying antisense oligomers

Fletcher, S., Bellgard, M., Price, L., Akkari, P. A., & Wilton, S. D. (2016) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2017.1250880 · Cited by 23

An update on RNA-targeting therapies for neuromuscular disorders

Jirka, S. M. G., & Aartsma-Rus, A. M. (2015) · Current Opinion in Neurology · doi:10.1097/wco.0000000000000235 · Cited by 22

Investigational therapies for the treatment of spinal muscular atrophy

Kaczmarek, A., Schneider, S. M., Wirth, B., & Rießland, M. (2015) · Expert Opinion on Investigational Drugs · doi:10.1517/13543784.2015.1038341 · Cited by 22

Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction

Hastings, M. L., & Jones, T. A. (2019) · Neurotherapeutics · doi:10.1007/s13311-019-00729-0 · Cited by 22

Transcriptional and Splicing Regulation of Spinal Muscular Atrophy Genes

Singh, N. N., Howell, M. D., & Singh, R. N. (2016) · Elsevier eBooks · doi:10.1016/b978-0-12-803685-3.00005-7 · Cited by 21

RNA control in pain: Blame it on the messenger

de la Peña, J. B., Song, J. J., & Campbell, Z. T. (2019) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1546 · Cited by 21

An Antisense Oligonucleotide Leads to Suppressed Transcription of Hdac2 and Long-Term Memory Enhancement

Poplawski, S. G., Garbett, K. A., McMahan, R. L., Kordasiewicz, H. B., Zhao, H. T., Kennedy, A. J., Goleva, S. B., Sanders, T. H., Motley, S. T., Swayze, E. E., et al. (2020) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2020.01.027 · Cited by 21

Oligonucleotide-based therapeutics for neurodegenerative disorders: Focus on antisense oligonucleotides

Mansour, H. M., & El-Khatib, A. S. (2025) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2025.177529 · Cited by 20

Knockdown of GADD34 in neonatal mutant SOD1 mice ameliorates ALS

Ghadge, G. D., Sonobe, Y., Camarena, A., Drigotas, C., Rigo, F., Ling, K., & Roos, R. P. (2019) · Neurobiology of Disease · doi:10.1016/j.nbd.2019.104702 · Cited by 22

Microinjection of antisense oligonucleotides into living mouse testis enables lncRNA function study

Chen, Z., Ling, L., Shi, X., Li, W., Zhai, H., Kang, Z., Zheng, B., Zhu, J., Ye, S., Wang, H., et al. (2021) · Cell & Bioscience · doi:10.1186/s13578-021-00717-y · Cited by 19

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Saoudi, A., Fergus, C., Gileadi, T. E., Montanaro, F., Morgan, J. E., Kelly, V. P., Tensorer, T., García, L., Vaillend, C., Muntoni, F., et al. (2023) · Cells · doi:10.3390/cells12060908 · Cited by 19

Pharmacodynamic Biomarkers for Emerging LRRK2 Therapeutics

Kelly, K., & West, A. B. (2020) · Frontiers in Neuroscience · doi:10.3389/fnins.2020.00807 · Cited by 19

Cognitive Impairment in Adult Patients with 5q-Associated Spinal Muscular Atrophy

Kizina, K., Akkaya, Y., Jokisch, D., Stolte, B., Totzeck, A., Rosales, J. M., Thimm, A., Bolz, S., Brakemeier, S., Pul, R., et al. (2021) · Brain Sciences · doi:10.3390/brainsci11091184 · Cited by 19

Self‐Assembled Antibody‐Oligonucleotide Conjugates for Targeted Delivery of Complementary Antisense Oligonucleotides

Zhou, L., Bi, J., Chang, S., Bai, Z., Yu, J., Wang, R., Li, Z., Zhang, X., Chou, J. J., & Pan, L. (2024) · Angewandte Chemie International Edition · doi:10.1002/anie.202415272 · Cited by 20

Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model

Berciano, M. T., Puente-Bedia, A., Medina-Samamé, A., Rodríguez-Rey, J. C., Calderó, J., Lafarga, M., & Tapia, O. (2020) · Scientific Reports · doi:10.1038/s41598-020-67569-3 · Cited by 19

Factors Influencing ADME Properties of Therapeutic Antisense Oligonucleotides: Physicochemical Characteristics and Beyond

Jiang, R., Hooshfar, S., Eno, M. R., Yun, C., Zimmermann, E. S., & Shinkyo, R. (2023) · Current Drug Metabolism · doi:10.2174/1389200224666230418092626 · Cited by 17

Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review

Nakevska, Z., & Yokota, T. (2023) · European Journal of Cell Biology · doi:10.1016/j.ejcb.2023.151326 · Cited by 18

Dosing, collection, and quality control issues in cerebrospinal fluid research using animal models

Barten, D. M., Cadelina, G. W., & Weed, M. R. (2017) · Handbook of clinical neurology · doi:10.1016/b978-0-12-804279-3.00004-6 · Cited by 17

Hydrogel-Assisted Antisense LNA Gapmer Delivery for In Situ Gene Silencing in Spinal Cord Injury

Moreno, P. M. D., Ferreira, A. K. D. S., Salvador, D., Rodrigues, M. T., Torrado, M., Carvalho, E. D., Tedebark, U., Sousa, M. M., Amaral, I. F., Wengel, J., et al. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.03.009 · Cited by 17

Evaluation of the effect of 2′-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice

Prakash, T. P., Yu, J., Kinberger, G. A., Low, A., Jackson, M., Rigo, F., Swayze, E. E., & Seth, P. P. (2018) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2018.10.011 · Cited by 17

RNP Assembly Defects in Spinal Muscular Atrophy

Price, P. L., Morderer, D., & Rossoll, W. (2018) · Advances in neurobiology · doi:10.1007/978-3-319-89689-2_6 · Cited by 15

The Medicinal Chemistry of RNase H-activating Antisense Oligonucleotides

Seth, P. P., & Swayze, E. E. (2019) · Royal Society of Chemistry eBooks · doi:10.1039/9781788015714-00032 · Cited by 16

Gene therapies for axonal neuropathies: Available strategies, successes to date, and what to target next

Morelli, K. H., Hatton, C. L., Harper, S. Q., & Burgess, R. W. (2020) · Brain Research · doi:10.1016/j.brainres.2020.146683 · Cited by 16

PEG-modification on the endo-position of an antisense oligonucleotide increases tumor accumulation via the EPR effect

Hagiwara, K., Kurihara, K., Honma, M., Yamamoto, J., & Shinohara, F. (2018) · Journal of Biomaterials Science Polymer Edition · doi:10.1080/09205063.2017.1422853 · Cited by 16

Novel regulators of PrP C expression as potential therapeutic targets in prion diseases

Baldeschi, A. C., Vanni, S., Zattoni, M., & Legname, G. (2020) · Expert Opinion on Therapeutic Targets · doi:10.1080/14728222.2020.1782384 · Cited by 16

Conditional Antisense Oligonucleotides Triggered by miRNA

Zhang, J., Sharma, R., Ryu, K., Shen, P., Salaita, K. S., & Jo, H. (2021) · ACS Chemical Biology · doi:10.1021/acschembio.1c00387 · Cited by 15

Antisense Oligonucleotide Modified with Disulfide Units Induces Efficient Exon Skipping in mdx Myotubes through Enhanced Membrane Permeability and Nucleus Internalization

Hiraoka, H., Shu, Z., Le, B. T., Masuda, K., Nakamoto, K., Lyu, F., Abe, N., Hashiya, F., Kimura, Y., Shimizu, Y., et al. (2021) · ChemBioChem · doi:10.1002/cbic.202100413 · Cited by 15

MiR-128-3p – a gray eminence of the human central nervous system

Kiel, K., Król, S. K., Bronisz, A., & Godlewski, J. (2024) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2024.102141 · Cited by 15

Correcting tau isoform ratios with a long-acting antisense oligonucleotide alleviates 4R-tauopathy phenotypes

Iwata‐Endo, K., Sahashi, K., Kawai, K., Fujioka, Y., Okada, Y., Watanabe, E., Iwade, N., Ishibashi, M., Mohammad, M., Aldoghachi, A. F., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102503 · Cited by 14

Advances in RNA therapeutics: Classes, innovations and clinical applications

Shahid, U. (2025) · The Journal of Precision Medicine Health and Disease · doi:10.1016/j.premed.2025.100016 · Cited by 18

Developing therapies for spinal muscular atrophy

Wertz, M. H., & Şahin, M. (2015) · Annals of the New York Academy of Sciences · doi:10.1111/nyas.12813 · Cited by 14

Evaluation of potential effects of Plastin 3 overexpression and low-dose SMN-antisense oligonucleotides on putative biomarkers in spinal muscular atrophy mice

Strathmann, E. A., Peters, M., Hosseinibarkooie, S., Rigo, F., Bennett, C. F., Zaworski, P. G., Chen, K. S., Nothnagel, M., & Wirth, B. (2018) · PLoS ONE · doi:10.1371/journal.pone.0203398 · Cited by 17

Aberrant Rna Homeostasis in Amyotrophic Lateral Sclerosis: Potential for New Therapeutic targets?

Donnelly, C. J., Grima, J. C., & Sattler, R. (2014) · Neurodegenerative Disease Management · doi:10.2217/nmt.14.36 · Cited by 15

Preclinical and Clinical Drug-metabolism, Pharmacokinetics and Safety of Therapeutic Oligonucleotides

Andersson, P. U., & den Besten, C. (2019) · Royal Society of Chemistry eBooks · doi:10.1039/9781788015714-00474 · Cited by 16

Pharmacology, pharmacokinetics, and metabolism of the DNA-decoy AYX1 for the prevention of acute and chronic post-surgical pain

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Systemic DNA/RNA heteroduplex oligonucleotide administration for regulating the gene expression of dorsal root ganglion and sciatic nerve

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Running title: Pharmacology of ISIS 396443 in the central nervous system Corresponding author:

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