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Browsing Tag

Gene Therapy

33 posts
GGenetics
A novel MCMDC2 variant causes meiotic arrest and non-obstructive azoospermia in a consanguineous Chinese family
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A novel MCMDC2 variant causes meiotic arrest and non-obstructive azoospermia in a consanguineous Chinese family

  • September 2, 2025
Cerván-Martín M, Castilla JA, Palomino-Morales RJ, Carmona FD. Genetic landscape of nonobstructive azoospermia and new perspectives for the…
GGenetics
MYH2-associated myopathy caused by novel compound heterozygous mutations: a case report and literature review
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MYH2-associated myopathy caused by novel compound heterozygous mutations: a case report and literature review

  • September 1, 2025
Morales PN, Coons AN, Koopman AJ, Patel S, Chase PB, Parvatiyar MS, et al. Post-translational modifications of vertebrate…
HHealth care
Expect health insurance prices to rise next year, brokers and experts say
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Expect health insurance prices to rise next year, brokers and experts say

  • August 26, 2025
Pricey prescriptions and nagging medical costs are swamping some insurers and employers now. Patients may start paying for…
GGenetics
A Japanese familial spastic paraplegia associated with a missense UBQLN2 variant
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A Japanese familial spastic paraplegia associated with a missense UBQLN2 variant

  • August 22, 2025
Statland JM, Barohn RJ, McVey AL, Katz JS, Dimachkie MM. Patterns of weakness, classification of motor neuron disease,…
GGenetics
Early lipid genetics: identification of common and rare genetic variants for lipid traits in Indian adolescents
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Early lipid genetics: identification of common and rare genetic variants for lipid traits in Indian adolescents

  • August 21, 2025
Heller DA, de Faire U, Pedersen NL, Dahlen G, McClearn GE. Genetic and environmental influences on serum lipid…
GGenetics
Baby KJ with his siblings
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Gene-Therapy Treatment Of Infant With Rare Genetic Condition Successful In World First » TwistedSifter

  • August 18, 2025
Pexels When baby KJ was born with a rare disease, which could cause a potentially fatal amount of…
GGenetics
Pathogenic ZNF319 variant disrupts nuclear localization and transcriptional regulation to cause a novel form of autosomal recessive leukodystrophy
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Pathogenic ZNF319 variant disrupts nuclear localization and transcriptional regulation to cause a novel form of autosomal recessive leukodystrophy

  • August 18, 2025
van der Knaap MS, Bugiani M. Leukodystrophies: a proposed classification system based on pathological changes and pathogenetic mechanisms.…
GGenetics
Expanding the genetic code in mammalian cells using pseudouridine modified codons
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New generation of CRISPR shows safer path to treating genetic diseases

  • August 17, 2025
A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases…
EEntrepreneurship
Join Sept. 18 with George Church and this year’s top biotech startups
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Join Sept. 18 with George Church and this year’s top biotech startups

  • August 13, 2025
I asked AI the oth­er day how many biotechs have been co-found­ed by Har­vard’s George Church. About 50…
GGenetics
Opus Genetics Announces VEGA-3 Phase 3 Trial Met its Primary Endpoint for Phentolamine Ophthalmic Solution 0.75% for the Treatment of Presbyopia
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Opus Genetics Announces Financial Results for Second Quarter 2025 and Provides Corporate Update

  • August 13, 2025
Opus Genetics, Inc. – Positive 12-month Phase 1/2 clinical data in adult cohort and early pediatric clinical data…
United States
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