Gene Therapy
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Genetic Time Travel: First Ancient Human Herpesvirus Genomes Reconstructed
Read more: Genetic Time Travel: First Ancient Human Herpesvirus Genomes ReconstructedThe findings trace the long history of HHV-6 integration into human chromosomes and suggest that HHV-6A lost this ability early on… trace the viruses’ evolution can now be traced over more than 2,500 years across Europe, using genomes from the 8th-6th century BCE until today.
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Rewiring the Mind: Restoring Missing Molecules in Down Syndrome
Read more: Rewiring the Mind: Restoring Missing Molecules in Down SyndromeScientists rewired Down syndrome brain circuits by restoring a missing molecule… potentially opening the way for new therapies that target the root cause of cognitive disruption.
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World’s First Base-Edited Therapy Achieves Deep Remission in Incurable Blood Cancer
Read more: World’s First Base-Edited Therapy Achieves Deep Remission in Incurable Blood CancerBase-editing technology, a more precise successor to CRISPR, has delivered deep remissions for 82% of patients with aggressive leukaemia.
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AI Predicts Disease from DNA Mutations
Read more: AI Predicts Disease from DNA MutationsAI breakthrough enables precise disease prediction from genetic mutations, paving the way for personalized medicine.
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FDA Approves First Precision Therapy for Aggressive BRCA2-Mutated Prostate Cancer
Read more: FDA Approves First Precision Therapy for Aggressive BRCA2-Mutated Prostate CancerA new FDA-approved dual-action pill targeting BRCA2 mutations significantly cuts prostate cancer progression risk by 54%.
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Regeneron and Tessera Partner on $150M Gene Editing Therapy for AATD
Read more: Regeneron and Tessera Partner on $150M Gene Editing Therapy for AATDThe partners will advance TSRA‑196, a one‑time gene editing therapy for alpha‑1 antitrypsin deficiency.
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FDA Approves Novartis’ Itvisma, Expanding Gene Therapy for SMA
Read more: FDA Approves Novartis’ Itvisma, Expanding Gene Therapy for SMAFDA greenlights intrathecal onasemnogene abeparvovec for SMA patients two years and older.
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Novel MIT Method Promises to Reduce Cost and Speed Up Gene Therapy Drug Manufacturing
Read more: Novel MIT Method Promises to Reduce Cost and Speed Up Gene Therapy Drug ManufacturingA selective crystallization method cuts gene therapy purification time by 90%, promising major cost reductions.
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Rare Neurological Disease Reversed in Animal Models via Gene Therapy
Read more: Rare Neurological Disease Reversed in Animal Models via Gene TherapyGene therapy in mice restores brain function and motor ability in a previously untreatable childhood neurological disease.
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CRISPR Gene Editing Reverses Chemotherapy Resistance in Lung Cancer
Read more: CRISPR Gene Editing Reverses Chemotherapy Resistance in Lung CancerKnockout of NRF2 via CRISPR resensitizes lung cancer cells to chemotherapy in preclinical models.