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Browsing Tag

Gene Therapy

10 posts
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…
GGenetics
Molecular genetics of myotonic dystrophy and the evolution of therapeutic approaches
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Molecular genetics of myotonic dystrophy and the evolution of therapeutic approaches

  • July 3, 2025
Nakamori, M & Takahashi, MP Myotonic dystrophy. In: Takeda S, Miyagoe-Suzuki Y, Mori-Yoshimura M, editors. Translational research in…
GGenetics
AAV gene therapy for autosomal recessive deafness 9: a single-arm trial
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AAV gene therapy for autosomal recessive deafness 9: a single-arm trial

  • July 2, 2025
Chang, Q. et al. Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in…
MMarkets
Custom Market Insights (Analysis, Outlook, Leaders, Report, Trends, Forecast, Segmentation, Growth Rate, Value, SWOT Analysis)
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Custom Market Insights (Analysis, Outlook, Leaders, Report, Trends, Forecast, Segmentation, Growth Rate, Value, SWOT Analysis)

  • June 25, 2025
Custom Market Insights [220+ Pages Latest Report] According to a market research study published by Custom Market Insights,…
United States
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