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0
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Specific and efficient RNA A-to-I editing through cleavage of an ADAR inhibitor.
(nature.com)
|
Li G
…
Chen J
Nature Biotechnology
2026-02-01
|
#gene therapy
#rna editing
#specificity
#adar
#base-editing
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0
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Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy.
(nature.com)
|
Buddle S
…
Breuer J
Nature Medicine
2026-02-01
|
#metagenomics
#sequencing
#gene therapy
#toxicity
#plasmid
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0
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Engineering a photoactivatable A-to-I RNA base editor for gene therapy in vivo.
(nature.com)
|
Li H
…
Li D
Nature Biotechnology
2026-02-01
|
#gene therapy
#rna editing
#cas13
#photoactivation
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0
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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
(nature.com)
|
Kwon JM
…
van der Pol WL
Nature Medicine
2026-02-01
|
#clinical trial
#gene therapy
#safety
#spinal muscular atrophy
|
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0
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Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.
(nature.com)
|
Proud CM
…
Finkel RS
Nature Medicine
2026-02-01
|
#clinical trial
#gene therapy
#spinal muscular atrophy
#motor function
|