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Health Sciences → Medicine → Genetics

Neurogenetic and Muscular Disorders Research

This cluster of papers focuses on the gene therapy, molecular genetics, and clinical management of Spinal Muscular Atrophy (SMA). It covers topics such as SMN1 and SMN2 genes, Nusinersen treatment, antisense oligonucleotide therapies, motor neuron pathology, and the role of survival motor neuron protein in SMA. The cluster also discusses potential therapeutic strategies, including gene replacement therapy and the use of small molecules to modulate SMN expression.

44.4K works worldwide465.8K citations
Spinal Muscular AtrophySMN1NusinersenGene Replacement TherapyMotor NeuronSMN2Antisense OligonucleotidesNeuromuscular JunctionSurvival Motor Neuron ProteinClinical Management

Journals publishing in this area

1
Neurology
ISSN 0028-38781,767 articles in this topic
520h-index
2Journal of the Neurological Sciences cover
Journal of the Neurological Sciences
ISSN 0022-510X1,005 articles in this topic
231h-index
3
Journal of Neurology Neurosurgery & Psychiatry
ISSN 0022-3050713 articles in this topic
335h-index
4Developmental Medicine & Child Neurology cover
Developmental Medicine & Child Neurology
ISSN 0012-1622624 articles in this topic
225h-index
5
Journal of Neurology
ISSN 0340-5354608 articles in this topic
203h-index
6Human Molecular Genetics cover
Human Molecular Genetics
ISSN 0964-6906592 articles in this topic
356h-index
7Annals of Neurology cover
Annals of Neurology
ISSN 0364-5134400 articles in this topic
435h-index
8
Journal of Medical Genetics
ISSN 0022-2593378 articles in this topic
246h-index
9
Acta Neuropathologica
ISSN 0001-6322327 articles in this topic
285h-index
10The Lancet Neurology cover
The Lancet Neurology
ISSN 1474-4422228 articles in this topic
451h-index