Dysregulation of microRNAs in spinal muscular atrophy and the response to antisense oligonucleotide therapy

2015 ◽  
Vol 25 ◽  
pp. S194
Author(s):  
H. Zhou ◽  
F. Catapano ◽  
I. Zaharieva ◽  
M. Scoto ◽  
J. Morgan ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

scholarly journals Wrangling RNA: Antisense oligonucleotides for neurological disorders

2019 ◽  
Vol 11 (511) ◽  
pp. eaay2069 ◽  
Author(s):  
Kevin Talbot ◽  
Matthew J. A. Wood
Keyword(s):  
Spinal Muscular Atrophy ◽  
Effective Treatment ◽  
Neurological Disorders ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

Effective treatment of spinal muscular atrophy with antisense oligonucleotide therapy opens the door to treating other neurological disorders with this approach.

scholarly journals Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

2020 ◽  
Vol 11 (3) ◽  
pp. 768-782 ◽  
Author(s):  
Haiyan Zhou ◽  
Jinhong Meng ◽  
Alberto Malerba ◽  
Francesco Catapano ◽  
Palittiya Sintusek ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Myostatin Inhibition ◽  
Antisense Oligonucleotide Therapy

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

2019 ◽  
Vol 105 (1) ◽  
pp. 221-230 ◽  
Author(s):  
Laura Torres-Benito ◽  
Svenja Schneider ◽  
Roman Rombo ◽  
Karen K. Ling ◽  
Vanessa Grysko ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

scholarly journals Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy

2016 ◽  
Vol 5 ◽  
pp. e331 ◽  
Author(s):  
Francesco Catapano ◽  
Irina Zaharieva ◽  
Mariacristina Scoto ◽  
Elena Marrosu ◽  
Jennifer Morgan ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Antisense Oligonucleotide Therapy

scholarly journals Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients

2021 ◽  
Vol 11 (3) ◽  
pp. 296
Author(s):  
Lars Hendrik Müschen ◽  
Alma Osmanovic ◽  
Camilla Binz ◽  
Konstantin F. Jendretzky ◽  
Gresa Ranxha ◽  
...  
Keyword(s):  
Cerebrospinal Fluid ◽  
Spinal Muscular Atrophy ◽  
Total Protein ◽  
Antisense Oligonucleotide ◽  
Muscular Atrophy ◽  
Neurological Diseases ◽  
Clinical Signs ◽  
Central Nervous System Disease ◽  
Oligoclonal Bands ◽  
System Disease

Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases. Changes in routine cerebrospinal fluid (CSF) parameters under nusinersen have only recently been described in adult SMA patients. We aimed to explore these findings in a real-world setting and to identify clinical and procedure-associated features that might impact CSF parameters. Routinely collected CSF parameters (leukocyte count, lactate, total protein, CSF/serum albumin quotient (QAlbumin), oligoclonal bands) of 28 adult SMA patients were examined for up to 22 months of nusinersen treatment. Total protein and QAlbumin values significantly increased in the first 10 months, independent of the administration procedure. By month 14, no further increases were detected. Two patients developed transient pleocytosis. In two cases, positive oligoclonal bands were found in the beginning and in four patients throughout the whole observation period. No clinical signs of inflammatory central nervous system disease were apparent. Our data confirm elevated CSF total protein and QAlbumin during nusinersen treatment. These alterations may be caused by both repeated lumbar punctures and the interval between procedures rather than by the medication itself. Generally, there were no severe alterations of CSF routine parameters. These results further underline the safety of nusinersen therapy.

scholarly journals Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

2019 ◽  
Vol 42 (1) ◽  
pp. 385-406 ◽  
Author(s):  
C. Frank Bennett ◽  
Adrian R. Krainer ◽  
Don W. Cleveland
Keyword(s):  
Neurodegenerative Diseases ◽  
Spinal Muscular Atrophy ◽  
Antisense Oligonucleotide ◽  
Antisense Oligonucleotides ◽  
Muscular Atrophy ◽  
Neurological Diseases ◽  
Great Promise ◽  
Disease Symptoms ◽  
Therapeutic Platform ◽  
Lateral Sclerosis

Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.

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