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0
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Intervening in aging and related diseases with gene therapy techniques.
(linkinghub.elsevier.com)
|
Jiang M
…
Liu GH
Cell Reports Medicine
2026-06-15
|
#gene therapy
#aging
#epigenetics
#genome editing
#telomeres
|
|
0
|
|
CSNK2B gene replacement rescues autism-related phenotypes and establishes translational EEG biomarkers.
(linkinghub.elsevier.com)
|
Ding C
…
Qiu Z
Cell Reports Medicine
2026-06-16
|
#gene therapy
#autism
#epilepsy
#biomarker
#neurodevelopmental
|
|
0
|
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Recombinant dimeric PICK1 peptide inhibitors for long-term relief of chronic pain by AAV therapeutics.
(linkinghub.elsevier.com)
|
Noes-Holt G
…
Sorensen AT
Cell Reports Medicine
2026-06-16
|
#gene therapy
#neuropathic pain
#proteomics
#peptide
#aav vectors
|
|
0
|
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Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia models.
(linkinghub.elsevier.com)
|
Pido-Lopez J
…
Sala A
Cell Reports Medicine
2026-06-16
|
#gene therapy
#cell therapy
#mitochondrial
#neurodegeneration
#hematopoietic
|
|
0
|
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Trial-ready external controls for gene therapy: The MATCH cohort in maple syrup urine disease.
(linkinghub.elsevier.com)
|
Brigatti KW
…
Strauss KA
Cell Reports Medicine
2026-06-16
|
#gene therapy
#metabolic disorder
#natural history
#clinical trial
#external controls
|
|
0
|
|
ABO: A 3D stroma-supported culture platform enabling full human B-lymphopoiesis for disease modeling and gene therapy development.
(linkinghub.elsevier.com)
|
Braams M
…
de Kivit S
Cell Reports Medicine
2026-06-18
|
#lymphopoiesis
#immunodeficiency
#gene therapy
#modeling
#bcr
|
|
0
|
|
Restoring the interplay between the endoplasmic reticulum and mitochondria by gene therapy improves Charcot-Marie-Tooth type 2A disease.
(pnas.org)
|
Tessier M
…
Bernard-Marissal N
PNAS
2026-06-23
|
#motor neuron
#gene therapy
#neuropathy
#mitochondria
#neurodegeneration
|
|
0
|
|
Patient-specific midbrain organoids with CRISPR correction recapitulate neuronopathic Gaucher disease phenotypes and enable evaluation of novel therapies.
(elifesciences.org)
|
Lin Y
…
Sun Y
eLife
2026-06-23
|
#organoid
#crispr
#lysosomal-storage-disorder
#gene therapy
#neurodegeneration
|
|
0
|
|
Cell type-specific contextualisation of the human phenome: towards the systematic treatment of all rare diseases.
(link.springer.com)
|
Schilder BM
…
Skene NG
Genome Medicine
2026-06-26
|
#transcriptomics
#rare-diseases
#phenotyping
#cell-type
#gene therapy
|
|
0
|
|
Re-administration of AAV-mediated gene therapy for OTOF-related deafness: a single-arm trial.
(nature.com)
|
Fan X
…
Shu Y
Nature Medicine
2026-06-26
|
#gene therapy
#deafness
#immunogenicity
#clinical trial
#aav
|
|
0
|
|
Retargeted serine integrases for one-step, precise integration of large DNA sequences in human cells.
(nature.com)
|
Fauser F
…
Miller JC
Nature Biotechnology
2026-06-29
|
#genome editing
#integrase
#directed evolution
#synthetic biology
#gene therapy
|
|
0
|
|
Targeted DNA triplex-forming oligonucleotide liposome for pulmonary fibrosis gene therapy.
(linkinghub.elsevier.com)
|
Xu J
…
Lu W
Cell Reports Medicine
2026-07-01
|
#fibrosis
#gene therapy
#oligonucleotide
#pulmonary
|
|
0
|
|
Inherited retinal degenerations: clinical phenotypes and emerging therapies.
(linkinghub.elsevier.com)
|
Uyhazi KE
…
Cideciyan AV
The Lancet
2026-07-06
|
#retinopathy
#genetics
#gene therapy
#cell therapy
#ophthalmology
|
|
0
|
|
Efficient targeting of human glial progenitor cells in vivo with engineered AAV vectors and glymphatic delivery.
(nature.com)
|
Cona A
…
Goldman SA
Nature Biotechnology
2026-07-08
|
#gene therapy
#glial
#aav
#cns
#neurodegeneration
|
|
0
|
|
Engineered ADARs enable precision A-to-G base editing of DNA.
(nature.com)
|
Im HW
…
Bae S
Nature Biotechnology
2026-07-10
|
#base-editing
#crispr
#gene therapy
#mutagenesis
|
|
0
|
|
Efficient in vivo cytosine base editing using virus-like particles with uracil DNA glycosylase inhibition.
(nature.com)
|
Zhu J
…
Chen J
Nature Biotechnology
2026-07-10
|
#base-editing
#gene therapy
#vlp
#delivery
|