Long Contact Times on AAV Affinity Resins Reshape Yield and Purity
Extended binding times on AAV affinity resins cause serotype-specific yield losses and VP3 enrichment while sharply improving host-cell impurity clearance ...
Tag: gene therapy
Prime Editing Moves Toward In Vivo Gene Correction for Genetic Diseases
A new review maps how prime editing, a CRISPR-derived tool capable in principle of correcting 89 percent of human pathogenic ...
Triplex-purified minicircle DNA restores frataxin in Friedreich’s ataxia cells and mice
A triplex-based purification method produced highly pure frataxin-expressing minicircle DNA vectors that restored frataxin protein in Friedreich's ataxia patient fibroblasts ...
Astrocytes Emerge as Unexpected Players in Rare Epileptic Disorder CDKL5
Patient-derived astrocytes and human brain slice models reveal water-transport, immune and cytoskeletal abnormalities linked to CDKL5 loss.
IGF-I Gene Therapy Reshapes the Proteome of Cartilage-Forming Stem Cells from Osteoarthritis Patients
A new proteomic study maps how rAAV-mediated IGF-I gene transfer alters protein expression in bone marrow stromal cells from osteoarthritis ...
Retina’s Master Support Cells Take Center Stage in Blinding Disease
A new review argues that Müller glia, the retina's dominant support cells, actively shape retinal disease progression and may offer ...
Gene Therapy for Duchenne Muscular Dystrophy Fails Key Trials, Review Finds
A systematic review and GRADE appraisal found that both phase 3 AAV-based shortened dystrophin gene therapy programs failed their primary ...
Gene Therapy Halves Key DNA Repair Signal in Primate Brains, Model Predicts Slower Huntington’s Progression
A new study combines a computational model of CAG repeat instability with preclinical testing of an AAV-delivered microRNA that reduced ...
Nine European Countries, Divergent Verdicts: How Health Technology Assessment Shapes Access to Advanced Therapies
A review of nine European countries finds that only six of 22 licensed ATMPs were assessed by all national HTA ...
Suppressor tRNAs restore mutant p53 and shrink tumours in mice
Suppressor tRNAs restored full-length p53 from nonsense-mutant TP53 and, delivered via lipid nanoparticles, cut tumour volume by up to 74 ...
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Mothers who receive childcare support from maternal grandparents show more parental warmth, finds NTU Singapore study
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University of Seville Breaks 120-Year-Old Mystery, Revises a Key Einstein Concept
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Bee body mass, pathogens and local climate influence heat tolerance
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Researchers record first-ever images and data of a shark experiencing a boat strike
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Groundbreaking Clinical Trial Reveals Lubiprostone Enhances Kidney Function
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