Results 111 to 120 of about 11,829 (290)

Noninvasive Focal Gene Delivery into the Cerebellum of Non‐Human Primates using Focused Ultrasound

Advanced Science, EarlyView.
Focal and non‐invasive viral vector delivery in non‐human primates remains a major challenge in translational neuroscience. Low‐intensity focused ultrasound was used to transiently open the blood–brain barrier and enable targeted gene delivery to the cerebellum.
Noelia Esteban‐García, José A. Pineda‐Pardo, Inés Trigo‐Damas, Marta Gutiérrez, Marta Castillo‐Ortiz, Megan Carrillo, Alejandro Reinares‐Sebastian, Víctor Medina‐Chavarrías, Itay Rachmilevitch, Masahiko Takada, José A. Obeso, Javier Blesa   +11 more
wiley   +1 more source

Recent advances in hereditary spinocerebellar ataxias.

, 2005
Contains fulltext : 48490.pdf (Publisher’s version ) (Open Access)In recent years, molecular genetic research has unraveled a major part of the genetic background of autosomal dominant and recessive spinocerebellar ataxias.
Warrenburg, B.P.C. van de, Kremer, Berry, van de Warrenburg, Bart P C, Sinke, R.J., Sinke, Richard J, Kremer, H.P.H.   +5 more
core   +2 more sources

CRPD frontiers in movement disorders Therapeutics: From evidence to treatment and applications

Clinical Parkinsonism & Related Disorders
The genetic ataxias have no cures and no proven ways to delay progression (no disease-modifying therapies). The acquired ataxias may have treatments that address the underlying cause and may slow or stop progression, but will not reverse damage already ...
Susan L. Perlman
doaj   +1 more source

Nucleic Acid Therapeutics for “Undruggable” Cancer Targets: Mechanisms, Challenges, and Prospects

Advanced Science, EarlyView.
Nucleic acid therapeutics bypass the structural limitations of conventional drugs by targeting mRNA rather than proteins. This review examines how antisense oligonucleotides, siRNAs, miRNAs, aptamers, and mRNA vaccines intervene against historically undruggable oncoproteins including Ras, MYC, and p53, highlighting mechanistic advances, delivery ...
Feng Xu, Ke Wang, Kaizhong Lu, Tianlun Hou, Yuan Chen, Xian Wang, Hongchuan Jin   +6 more
wiley   +1 more source

Past, present and future therapeutics for cerebellar ataxias.

, 2010
Cerebellar ataxias are a group of disabling neurological disorders. Patients exhibit a cerebellar syndrome and can also present with extra-cerebellar deficits, namely pigmentary retinopathy, extrapyramidal movement disorders, pyramidal signs, cortical ...
Manto, Mario, Marmolino, Daniele
core   +1 more source

Accelerating Biosensor Discovery: A Computationally‐Driven Pipeline for Microplastics Monitoring

Advanced Intelligent Discovery, EarlyView.
A computationally guided pipeline unites molecular simulation, synthetic biology, electrochemical engineering, and machine learning to accelerate biosensor discovery. A Bacillus anthracis carbohydrate‐binding module is used to develop a high‐performance micro‐ and nanoplastics sensor with greatly reduced error and variability.
Gabriel X. Pereira, Marcos de C. Leite, Pedro H. M. Zanineli, Juliana N. Y. Costa, Débora M. Cunha, Sophia P. Serra, Pedro H. Sophia, Flávio M. Shimizu, Yasmin W. Moura, Célio C. Oliveira, Gabriel R. Schleder, Gabriela F. Persinoti, Leandro Merces, Renato S. Lima   +13 more
wiley   +1 more source

Real‐World Evaluation of Talquetamab for the Treatment of Relapsed/Refractory Multiple Myeloma (RRMM): An International Myeloma Working Group Immunotherapy Registry Real‐World Analysis

American Journal of Hematology, EarlyView.
ABSTRACT Talquetamab is a GPRC5D × CD3 bispecific antibody approved for relapsed/refractory multiple myeloma (RRMM). This is the first real‐world study to comprehensively report muco‐cutaneous toxicities, infections, and efficacy of talquetamab in 151 patients across five countries with the longest follow‐up reported enabling better efficacy and ...
Murali Janakiram, Carlyn R. Tan, Hira Mian, Chiung‐Yu Huang, Rakesh Popat, Joaquín Martínez‐Lopez, Efsthathios Kastritis, Wee‐Joo Chng, Prashant Kapoor, Mrugakshi Dave, Susan Bal, Laurent Garderet, Andre De Menezes Silva Corraes, Christine Riedhammer, Torsten Steinbrunn, Magdalene Corona, Chandramouli Nagarajan, Hermann Einsele, Tom Martin, Amrita Krishnan, Yi Lin, Nadine Abdullah, Niels Abildgaard, Ingerid Abrahamsen, Melissa Alsina, Kenneth Anderson, Hervé Avet‐Loiseau, Ashraf Badros, Nizar Jacques Bahlis, Susan Bal, Rahul Banerjee, Dina Ben‐Yehuda, Jesús G. Berdeja, Michele Cavo, Ajai Chari, Wen‐Ming Chen, Marta Chesi, Wee‐Joo Chng, Yael Cohen, Adam Cohen, Gordon Cook, Luciano Costa, Andrew Cowan, Carlos Fernández de Larrea, Benjamin Derman, Juan Du, Hermann Einsele, Gösta Gahrton, Francesca Gay, Sergio A. Giralt, Simon Harrison, J Jean‐Luc Harousseau, Joy Ho, Doris Hansen, Maung Myo Htut, Andrzej J. Jakubowiak, Murali Janakiram, Prashant Kapoor, Efstathios Kastritis, Jonathan Kaufman, Kihyun Kim, Amrita Krishnan, Shaji Kumar, Chara Kyriakou, Xavier Leleu, Yi Lin, Sagar Lonial, Matthew frigault, Johan L. Lund, Elias Mai, Angelo Maiolino, Elias Mai, Angelo Maiolino, Sireesha Asoori Maringanti, Thomas Martin, Joaquin Martinez‐Lopez, María‐Victoria Mateos, Giampaolo Merlini, Hira Mian, Joseph Mikhael, Monique Minnema, Philippe Moreau, Gareth Morgan, Nikhil Munshi, Omar Nadeem, Chandramouli Nagarajan, Ajay Nooka, Michael O'Dwyer, Rimke Oostvogel, Alberto Orfao, Paula Rodriguez Otero, Darren Pan, Krina Patel, Rashesh Popat, Noemí Puig   +94 more
wiley   +1 more source

A novel approach for treating cerebellar ataxias.

, 2008
The terminology of cerebellar ataxias encompasses a variety of sporadic and inherited debilitating diseases. Patients exhibit disabling deficits such as dysmetria, kinetic tremor and ataxia of stance/gait. We are currently lacking effective treatments in
Oulad Ben Taib, Nordeyn, Manto, Mario
core   +1 more source

Atypical Clinical Course of Griscelli Syndrome Type 2 With Primarily Neurologic Presentation and Adult‐Onset in a 46‐Year‐Old Male

American Journal of Medical Genetics Part A, EarlyView.
ABSTRACT Griscelli Syndrome Type 2 (GS2) is a rare autosomal recessive disorder caused by pathogenic mutations in the RAB27A gene. Typically, it is characterized by cutaneous hypopigmentation, immunodeficiency, with or without neurological abnormalities secondary to hemophagocytic lymphohistiocytosis (HLH). Without treatment, GS2 often results in fatal
Dzhoy Papingi, Michael Kutsche, Helena Lichtenfeld, Fanny Kortüm, Angela Abicht, Laura Herrmann, Theresia Herget   +6 more
wiley   +1 more source

Comorbidities and clinical severity of COVID-19: systematic review and meta-analysis

Revista Habanera de Ciencias Médicas, 2020
Introduction: The recent emergence of COVID-19 has become a serious global health problem. The identification of comorbidities associated with the clinical severity in COVID-19 patients is of paramount significance for the appropriate therapeutic ...
Thais María Plasencia-Urizarri, Raúl Aguilera-Rodríguez, Luis Enrique Almaguer Mederos   +2 more
doaj