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Browsing Tag

Gene Therapy

25 posts
GGenetics
Targeted in vivo gene integration of a secretion-enabled GLP-1 receptor agonist reverses diet-induced non-genetic obesity and pre-diabetes
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Targeted in vivo gene integration of a secretion-enabled GLP-1 receptor agonist reverses diet-induced non-genetic obesity and pre-diabetes

  • 18 July 2025
Plasmid construction The Exe4 gene was codon-optimized using the GeneSmart codon optimization tool (https://www.genscript.com/gensmart-free-gene-codon-optimization.html) based on the amino…
GGenetics
Gene therapy brings back hearing in young and adult deaf patients
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Gene therapy brings back hearing in young and adult deaf patients

  • 4 July 2025
Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new…
GGenetics
Review of 40 genes causing congenital myasthenic syndromes
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Review of 40 genes causing congenital myasthenic syndromes

  • 18 June 2025
Ohno K, Ohkawara B, Shen XM, Selcen D, Engel AG. Clinical and pathologic features of congenital myasthenic syndromes…
GGenetics
Integration of MRI radiomics and germline genetics to predict the IDH mutation status of gliomas
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Integration of MRI radiomics and germline genetics to predict the IDH mutation status of gliomas

  • 16 June 2025
An overview of the study design and analysis is provided in Fig. 1. Demographic and clinical characteristics of…
GGenetics
SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines
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SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines

  • 11 June 2025
Financing co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund, as…
GGenetics
The impact of public insurance on RRSO for HBOC in Japan: a nationwide data study
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The impact of public insurance on RRSO for HBOC in Japan: a nationwide data study

  • 21 May 2025
JOHBOC Registration Committee: Masami Arai (Department of Clinical Genetics, Juntendo University, Graduate School of Medicine, Tokyo, Japan), Seigo…
GGenetics
Scientists call for 10-year ban on CRISPR for germline gene editing
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Scientists call for 10-year ban on CRISPR for germline gene editing

  • 19 May 2025
Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT…
GGenetics
ACMG secondary findings in the Brazilian rare genomes project: insights from 5402 genome sequencing
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ACMG secondary findings in the Brazilian rare genomes project: insights from 5402 genome sequencing

  • 19 May 2025
Green RC, Berg JS, Grody WW, Kalia SS, Korf BR, Martin CL, et al. ACMG recommendations for reporting…
GGenetics
Custom Gene Editing Helps Baby With Rare Condition | Health
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Custom Gene Editing Helps Baby With Rare Condition | Health

  • 17 May 2025
Key Takeaways A baby with a rare genetic disorder received a custom gene editing treatment Docs used a…
GGenetics
Reclassification of variants of uncertain significance in neonatal genetic diseases: implications from a clinician’s perspective
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Reclassification of variants of uncertain significance in neonatal genetic diseases: implications from a clinician’s perspective

  • 12 May 2025
Yang L, Liu X, Li Z, Zhang P, Wu B, Wang H, et al. Genetic aetiology of early…
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