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14 results on '"Gabbeta, Vijayalakshmi"'

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1. Development of an oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia

2. A deep learning approach to identify gene targets of a therapeutic for human splicing disorders

3. Small molecule splicing modifiers with systemic HTT-lowering activity

4. Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment

5. Development of a novel oral treatment that rescues gait ataxia and retinal degeneration in a phenotypic mouse model of familial dysautonomia

6. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

7. MOTOR NEURON DISEASE: SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

8. A deep learning approach to identify new gene targets of a novel therapeutic for human splicing disorders

9. ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia

10. Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy

14. Differential Binding of an SRF/NK-2/MEF2 Transcription Factor Complex in Normal VersusNeoplastic Smooth Muscle Tissues*

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