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0
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Recombinant dimeric PICK1 peptide inhibitors for long-term relief of chronic pain by AAV therapeutics.
(linkinghub.elsevier.com)
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Noes-Holt G
…
Sorensen AT
Cell Reports Medicine
2026-06-16
|
#proteomics
#gene therapy
#peptide
#neuropathic pain
#aav vectors
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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
|
#mitochondrial
#neurodegeneration
#gene therapy
#cell therapy
#hematopoietic
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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
|
#clinical trial
#gene therapy
#metabolic disorder
#natural history
#external controls
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0
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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
#biomarker
#epilepsy
#neurodevelopmental
#autism
|