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Browsing Tag

Gene Therapy

33 posts
GGenetics
Genetic insights into the origin, admixture, and migration of the early Austronesian peoples
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Genetic insights into the origin, admixture, and migration of the early Austronesian peoples

  • August 7, 2025
Shutler R, Marck JC. On the dispersal of the Austronesian horticulturalists. Archaeology Phys Anthropol Ocean. 1975;10:81–113. Google Scholar …
GGenetics
When ganglioside pathways go awry: congenital disorders and experimental insights
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When ganglioside pathways go awry: congenital disorders and experimental insights

  • July 31, 2025
Thudichum JLW. A treatise on the chemical constitution of the brain: Bailliere, Tindall, and Cox, London; 1884. Chiricozzi…
BBusiness
FDA vaccine chief Vinay Prasad leaves following criticism from Laura Loomer
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FDA vaccine chief Vinay Prasad leaves following criticism from Laura Loomer

  • July 30, 2025
WASHINGTON (AP) — The Food and Drug Administration’s polarizing vaccine chief is leaving the agency after a brief…
GGenetics
Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation
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Rocket’s PKP2-Arrhythmogenic Cardiomyopathy Gene Therapy RP-A601 Snags RMAT Designation

  • July 29, 2025
Rocket Pharmaceuticals’ RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related Arrhythmogenic Cardiomyopathy…
GGenetics
Breakthrough gene therapy reverses genetic deafness across age groups
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Breakthrough gene therapy reverses genetic deafness across age groups

  • July 23, 2025
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to…
GGenetics
Molecular genetics of J-domain protein-related chaperonopathies in skeletal muscle
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Molecular genetics of J-domain protein-related chaperonopathies in skeletal muscle

  • July 22, 2025
Kampinga HH, Andreasson C, Barducci A, Cheetham ME, Cyr D, Emanuelsson C, et al. Function, evolution, and structure…
GGenetics
Sequencing validates deep learning models for EHR-based detection of Noonan syndrome in pediatric patients
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Sequencing validates deep learning models for EHR-based detection of Noonan syndrome in pediatric patients

  • July 21, 2025
Cohort characteristics and model risk score distribution The study cohort comprised 92,493 patients enrolled in the DT Biobank…
FFort Worth
Nanoscope Begins FDA Submission for Breakthrough Retinitis Pigmentosa Therapy
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Nanoscope Begins FDA Submission for Breakthrough Retinitis Pigmentosa Therapy

  • July 14, 2025
Dallas-based Nanoscope Therapeutics has taken a major step toward offering new hope to people who are losing their…
HHealth
These Genetically Modified Mice Make Their Own 'Ozempic'
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These Genetically Modified Mice Make Their Own ‘Ozempic’

  • July 9, 2025
The popularity of Ozempic and Wegovy as diabetes and weight loss medications has skyrocketed. But how far would…
GGenetics
Genetic and oncogenic features of RASGRF fusions
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Genetic and oncogenic features of RASGRF fusions

  • July 5, 2025
Prevalence of RASGRF fusions in solid tumor malignancies We previously reported three RASGRF1 fusions: (1) an OCLN–RASGRF1 fusion…
United States
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