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| http://dx.doi.org/10.1111/j.1601-5215.2010.00484.x | DOI Listing |
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RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington's disease models.
Mol Neurodegener
January 2025
Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), School of Medicine, GHM Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.
Background: HD is a devastating neurodegenerative disorder caused by the expansion of CAG repeats in the HTT. Silencing the expression of mutated proteins is a therapeutic direction to rescue HD patients, and recent advances in gene editing technology such as CRISPR/CasRx have opened up new avenues for therapeutic intervention.
Methods: The CRISPR/CasRx system was employed to target human HTT exon 1, resulting in an efficient knockdown of HTT mRNA.
Effect of Triheptanoin on Caudate Atrophy and Motor Scores in Patients With Early-Stage Huntington Disease: A Phase II Study.
Neurology
January 2025
APHP- Salpêtrière Hospital, DMU BioGem, CNRS, INSERM, Paris Brain Institute, Sorbonne University.
Background And Objectives: Brain energy deficiency occurs at the early stage of Huntington disease (HD). Triheptanoin, a drug that targets the Krebs cycle, can restore a normal brain energetic profile in patients with HD. In this study, we aimed at assessing its efficacy on clinical and neuroimaging structural measures in HD.
View Article and Find Full Text PDFAdult-Onset Myoclonus in a Large Urban Inpatient Setting: A Retrospective Cohort Study.
Tremor Other Hyperkinet Mov (N Y)
January 2025
Department of Neurology, New York Presbyterian Brooklyn Methodist Hospital, Brooklyn, NY, US.
Background: Myoclonus is a hyperkinetic movement with various attributable etiologies, semiologies, and treatment outcomes. To our knowledge, few studies investigated adult-onset myoclonus in an inpatient setting.
Methods: We retrospectively reviewed charts of adult inpatients with myoclonus at New York Presbyterian Brooklyn Methodist Hospital between 2011 and 2021.
Longitudinal Serotonergic and Dopaminergic Binding: Impact on Parkinson's Disease Progression and Levodopa Dyskinesia.
J Neuroimaging
January 2025
Department of Nuclear Medicine, Seoul National University Bundang Hospital, Seongnam-si, Republic of Korea.
Background And Purpose: We investigated the relationship between serotonergic and dopaminergic specific binding transporter ratios (SBRs) over 4 years in Parkinson's disease (PD) patients. We assessed serotonergic innervation's potential compensatory role for dopaminergic denervation, association with PD symptoms, and involvement in the development of levodopa-induced dyskinesia (LID).
Methods: SBRs of the midbrain and striatum were evaluated from [I-123] N-ω-fluoropropyl-2β-carbomethoxy-3β-(4-iodophenyl)nortropane SPECT images at baseline and after 4 years.
Intravenous lidocaine decreased oxygen-desaturation episodes induced by propofol-based sedation for gastrointestinal endoscopy procedures: a prospective, randomized, controlled trial.
BMC Anesthesiol
January 2025
Department of Anesthesiology, Beijing Friendship Hospital, Capital Medical University, No.95 Yongan Road, Beijing, Xicheng District, 100050, China.
Background: As a popularly used analgesic adjuvant, intravenous (IV) lidocaine could reduce the consumption of propofol in painless gastrointestinal (GI) endoscopy. However, whether IV lidocaine could affect the incidence of oxygen-desaturation episodes (ODE) during painless GI endoscopy is still unknown. Therefore, we tested the hypothesis that IV lidocaine could decrease the incidence of propofol-induced ODE and involuntary movements in patients during GI endoscopy.
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