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1. New Findings from State University of New York (SUNY) Buffalo Describe Advances in Gene Therapy (The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome)

2. Research from Nationwide Children's Hospital Research Institute Has Provided New Data on Gene Therapy (In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders)

3. Researchers from Pediatrics and Rare Diseases Group Detail New Studies and Findings in the Area of Gene Therapy (Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease)

4. Researchers at Pediatrics and Rare Diseases Group Target Gene Therapy (Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8 mnd ...)

5. Pediatrics and Rare Diseases Group Researchers Detail Findings in Gene Therapy (Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice)

6. Recent Findings in Gene Therapy Described by Researchers from Sanford Research (Aav9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities In a Mouse Model of Cln8-batten Disease)

7. Research Reports from Fundacion Cardiovascular de Colombia Provide New Insights into Gene Therapy (Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart ...)

8. New Biology Research from University of Lisbon Discussed (Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes)

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