A systematic review indicates that while engineered exosomes offer unique biological delivery advantages for cancer therapy, their clinical translation remains ...
A novel engineered adeno-associated virus demonstrates choroid plexus tropism across species and delivers targeted CRISPR gene editing in mice while ...
Researchers found that Dhx36 modulates a promoter G-quadruplex to activate Alpl transcription and restore bone mass in osteoporotic mice.
Researchers propose adapting hemophilia comprehensive care principles to establish multidisciplinary care centers and national registries for hereditary angioedema.
A new review in Materials Today Bio explores how gene-activated matrices use nanomedicine to sustain bone repair by integrating scaffold ...
A 2026 review in Acta Epileptologica details how KCNT1 gene mutations alter Slack channel function, leading to various forms of ...
A pooled analysis of four clinical trials evaluating delandistrogene moxeparvovec in predominantly young, ambulatory patients with Duchenne muscular dystrophy demonstrated ...
A comprehensive review analyzes how microneedle arrays overcome biological barriers to deliver nucleic acids and gene-editing platforms directly into local ...
A subretinally delivered gene therapy vector incorporating a native untranslated regulatory region achieved fifteen months of sustained structural and functional ...
A preclinical study demonstrates that local rapamycin microsphere delivery enables durable lentiviral gene therapy for Dent disease type 1 in ...
A density gradient equilibrium analytical ultracentrifugation method using rubidium chloride achieved high-resolution separation and accurate quantification of the two highly ...
Extended binding times on AAV affinity resins cause serotype-specific yield losses and VP3 enrichment while sharply improving host-cell impurity clearance ...
A new review maps how prime editing, a CRISPR-derived tool capable in principle of correcting 89 percent of human pathogenic ...
A triplex-based purification method produced highly pure frataxin-expressing minicircle DNA vectors that restored frataxin protein in Friedreich's ataxia patient fibroblasts ...
Patient-derived astrocytes and human brain slice models reveal water-transport, immune and cytoskeletal abnormalities linked to CDKL5 loss.
A new proteomic study maps how rAAV-mediated IGF-I gene transfer alters protein expression in bone marrow stromal cells from osteoarthritis ...
A new review argues that Müller glia, the retina's dominant support cells, actively shape retinal disease progression and may offer ...
A systematic review and GRADE appraisal found that both phase 3 AAV-based shortened dystrophin gene therapy programs failed their primary ...
A new study combines a computational model of CAG repeat instability with preclinical testing of an AAV-delivered microRNA that reduced ...
A review of nine European countries finds that only six of 22 licensed ATMPs were assessed by all national HTA ...
