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Biodistribution of radioactively labeled splice-modulating antisense oligonucleotides after intracerebroventricular and intrathecal injection in mice. Nucleic Acid Ther. 34 (1), 26-34 (2024).","journal-title":"Nucleic Acid Ther"},{"issue":"19","key":"ref54","doi-asserted-by":"crossref","first-page":"3051","DOI":"10.1021\/acschemneuro.0c00409","type":"journal-article","article-title":"Comparative intracerebroventricular and intrathecal administration of a nanomolar macrocyclic melanocortin receptor agonist MDE6-5-2c (c[Pro-His-DPhe-Arg-Trp-Dap-Ala-DPro]) decreases food intake in mice","volume":"11","author":"Adank","year":"2020","unstructured":"Adank, D. N. et al. Comparative intracerebroventricular and intrathecal administration of a nanomolar macrocyclic melanocortin receptor agonist MDE6-5-2c (c[Pro-His-DPhe-Arg-Trp-Dap-Ala-DPro]) decreases food intake in mice. ACS Chem Neurosci. 11 (19), 3051-3063 (2020).","journal-title":"ACS Chem Neurosci"},{"issue":"3","key":"ref55","doi-asserted-by":"crossref","first-page":"1242","DOI":"10.4081\/cp.2020.1242","type":"journal-article","article-title":"Multiple doses of cell therapy and neurorehabilitation in amyotrophic lateral sclerosis: a case report","volume":"10","author":"Sharma","year":"2020","unstructured":"Sharma, A. et al. Multiple doses of cell therapy and neurorehabilitation in amyotrophic lateral sclerosis: a case report. Clin Pract. 10 (3), 1242 (2020).","journal-title":"Clin Pract"},{"key":"ref56","doi-asserted-by":"crossref","first-page":"693309","DOI":"10.3389\/fneur.2021.693309","type":"journal-article","article-title":"Gene therapy overexpressing neuregulin 1 type I in combination with neuregulin 1 type III promotes functional improvement in the SOD1(G93A) ALS mice","volume":"12","author":"Modol-Caballero","year":"2021","unstructured":"Modol-Caballero, G. et al. Gene therapy overexpressing neuregulin 1 type I in combination with neuregulin 1 type III promotes functional improvement in the SOD1(G93A) ALS mice. 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