42 results on '"Nelvagal, Hemanth R."'
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2. Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
3. Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a [Cln2.sup.R207X] mouse model
4. GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice
5. Synergistic effects of treating the spinal cord and brain in CLN1 disease
6. mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases.
7. Pathomechanisms in the neuronal ceroid lipofuscinoses
8. Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor
9. Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
10. Bowel dysmotility and enteric neuron degeneration in lysosomal storage disease mice is prevented by gene therapy
11. Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease
12. Spontaneous seizures associated with cortical interneuron loss in mice are ameliorated via gene therapy
13. Top-down and bottom-up propagation of disease in the neuronal ceroid lipofuscinoses
14. Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB
15. Glial Dysfunction and Its Contribution to the Pathogenesis of the Neuronal Ceroid Lipofuscinoses
16. Cortical interneuron loss and seizure generation as novel clinically relevant disease phenotypes in Cln2R207X mice
17. Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures
18. Spinal cord pathology in murine Sanfilippo syndrome type B
19. Efficacy of recombinant human PPT1 enzyme replacement therapy in mouse and sheep models of CLN1 disease
20. Amelioration of enteric nervous system defects via gene therapy in CLN1 disease mice
21. Correction: Corrigendum: mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases
22. Quantifying storage material accumulation in tissue sections
23. Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndrome
24. Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndrome
25. Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
26. Devising effective enzyme replacement therapy for infantile onset neuronal ceroid lipofuscinosis (CLN1 disease)
27. Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye
28. Immune cells perturb axons and impair neuronal survival in a mouse model of infantile neuronal ceroid lipofuscinosis
29. Early gait abnormalities relate to brainstem and spinal cord pathology in CLN1 disease
30. Translating preclinical models of neuronal ceroid lipofuscinosis: progress and prospects
31. Progress toward Fulfilling the Potential of Immunomodulation in Childhood Neurodegeneration?
32. 27. Therapeutic Efficacy of Intracranial and Intrathecal AAV2/9-PPT1 in Infantile Batten Disease
33. Towards a new understanding of NCL pathogenesis
34. Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis
35. Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene
36. Corrigendum: mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases.
37. Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model.
38. Spontaneous seizures associated with cortical interneuron loss in Cln2R207Xmice are ameliorated via gene therapy.
39. GABAergic interneurons contribute to the fatal seizure phenotype of CLN2 disease mice.
40. Neuronal deletion of the circadian clock gene Bmal1 induces cell-autonomous dopaminergic neurodegeneration.
41. Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
42. Quantifying storage material accumulation in tissue sections.
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