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6
|
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BIN1 overexpression rescues cardiac but not skeletal muscle defects in a mouse model of caveolinopathy.
(academic.oup.com)
|
Moncheaux A
…
Laporte J
Human Molecular Genetics
2026-08-25
|
#caveolinopathy
#cardiomyopathy
#myopathy
#muscle
#gene therapy
|
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6
|
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HMGB1-targeted anti-inflammatory rBox A gene therapy for viral airway infections and systemic inflammatory disease models.
(journals.asm.org)
|
Shirey KA
…
Vogel SN
mBio
2026-08-24
|
#immunotherapy
#inflammation
#gene therapy
#sepsis
#viral infection
|
|
3
|
|
Carboxyl-terminal blockade of sortilin binding enhances progranulin gene therapy in a mouse model of frontotemporal dementia.
(science.org)
|
Kashyap SN
…
Roberson ED
Science Translational Medicine
2026-08-19
|
#gene therapy
#neurodegeneration
#frontotemporal-dementia
#neuroinflammation
#protein design
|
|
2
|
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Dual-gene regulation of NGF overexpression and P75NTR knockdown enhances BMSC osteogenesis by suppressing pyroptosis.
(linkinghub.elsevier.com)
|
Ji Y
…
Bei C
iScience
2026-08-21
|
#osteogenesis
#stem cell
#bone-regeneration
#pyroptosis
#gene therapy
|
|
0
|
|
Ultrasound-guided renal artery administration of angiopoietin-1 RNA therapy slows the progression of preclinical WT1 glomerular disease.
(science.org)
|
Malik SN
…
Long DA
Science Translational Medicine
2026-08-12
|
#gene therapy
#glomerulonephritis
#kidney disease
#nanomedicine
#rna
|
|
0
|
|
GNPs-pIL-4 reprograms macrophage polarization and activates the OSM/GSNOR/ENG axis to improve angiogenesis in ischemic limbs.
(science.org)
|
Zhang P
…
Feng J
Science Advances
2026-08-12
|
#angiogenesis
#gene therapy
#macrophage
#nanoparticles
#ischemia
|
|
0
|
|
TIGRa: An ultra-compact programmable activator enabling multiplexed and efficient gene regulation.
(linkinghub.elsevier.com)
|
Liu Z
…
Sun Y
Cell Stem Cell
2026-08-10
|
#activation
#crispr
#gene therapy
#transcriptional-regulation
#synthetic biology
|
|
0
|
|
eVLP compound delivery breaks the prime editing efficiency ceiling.
(linkinghub.elsevier.com)
|
Wang MR
…
Sanchez-Rivera FJ
Cell Genomics
2026-08-12
|
#base-editing
#gene therapy
#delivery
#virus-like-particles
|
|
0
|
|
AAV-NRF2 protects retinal and choroidal vasculature in a GDF15-dependent manner in an oxidative damage model of AMD.
(pnas.org)
|
Wang S
…
Cepko CL
PNAS
2026-08-11
|
#gene therapy
#oxidative stress
#amd
#neuroprotection
#transcriptomics
|
|
0
|
|
Rapid resolution of variants of uncertain significance (VUS). A complementary role for zebrafish in the era of multi-million-dollar therapies.
(nature.com)
|
Giacomotto J
npj Genomic Medicine
2026-08-10
|
#variants
#pathogenicity
#precision medicine
#gene therapy
|
|
0
|
|
An ER stress-responsive RNA rheostat for programmable gene and mRNA therapies.
(linkinghub.elsevier.com)
|
Ogurlu R
…
Asokan A
Cell Chemical Biology
2026-08-05
|
#gene therapy
#mrna therapy
#protein design
#stress response
#synthetic biology
|
|
0
|
|
Temporal vision adaptation and restoration in achromatopsia.
(linkinghub.elsevier.com)
|
McKyton A
…
Levin N
iScience
2026-08-05
|
#achromatopsia
#vision
#gene therapy
#temporal-sensitivity
|
|
0
|
|
AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne Syndrome.
(jci.org)
|
Batista AR
…
Sena-Esteves M
Journal of Clinical Investigation
2026-07-30
|
#gene therapy
#cockayne-syndrome
#neurodegeneration
#aav
#dna repair
|
|
0
|
|
Gene therapy for hypertrophic cardiomyopathy: From feasibility to a fieldwide framework.
(linkinghub.elsevier.com)
|
Desai MY
…
Braunwald E
Cell Med
2026-07-30
|
#gene therapy
#cardiomyopathy
#vector
#immunosuppression
#clinical translation
|
|
0
|
|
Rewriting the sarcomere: Gene therapy approaches for hypertrophic cardiomyopathy from bench to bedside.
(linkinghub.elsevier.com)
|
Keen SK
…
Desai MY
Cell Med
2026-07-29
|
#gene therapy
#cardiomyopathy
#sarcomere
#genome editing
#cardiac
|