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Papers, explained in our own words

Every entry summarises what the study set out to test, what it found and why it changes how we design delivery systems. Browse research and reviews or search the collection.

706 articles

Showing 1–3 of 3 articles · Keyword: Adeno-associated Virus Clear keyword

Current cardiology reports2026ReviewNon-viral Gene Delivery

1. Viral Gene Therapy for Danon Disease: From LAMP2 Biology to Clinical Translation

Gunes Ea, Patel U, Iyer S, Taub A, Lee-Guest E, Cline S, Et Al.

Danon disease is a rare, highly penetrant and morbid X-linked cardioskeletal myopathy caused by loss-of-function variants in the lysosome-associated membrane protein 2 (LAMP2) gene. We review the rationale for LAMP2B gene replacement, the basis for adeno-associated virus serotype 9 (AAV9) vector selection, the…

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Human gene therapy2026ResearchNon-viral Gene Delivery

2. AAV6 Preferentially Transduces Human Choroid Plexus Epithelium in Cerebrospinal Fluid

Bacha T, Ren Y, Fan J, Mcmahon Mm, Panigrahi A, Warner D, Et Al.

Adeno-associated virus (AAV) vectors for gene delivery have demonstrated efficacy in treating genetic and acquired human diseases. For the treatment of neurological disorders, AAV delivery via the cerebrospinal fluid (CSF) enables transduction of therapeutic genes to cellular targets in the nervous system, bypassing…

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Frontiers in cellular neuroscience2026ResearchNon-viral Gene Delivery

3. Adeno-associated virus gene delivery targeting corneal-projecting trigeminal ganglion neurons

Reynolds Ss, Rasheed Mz, Niyonkuru K, Parikh S, Whyland K, Merrill Bm, Et Al.

Corneal innervation is essential for ocular surface homeostasis and protection, yet methods for selectively labeling and manipulating cornea-projecting neurons remain limited. Conventional retrograde tracers can identify cornea-projecting cell bodies in the trigeminal ganglion (TG), but they do not enable the…

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