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24
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Optogenetic Therapy for Restoring Aspects of Visual Function.
(nejm.org)
|
Sahel JA
…
Roska B
New England Journal of Medicine
2026-10-08
|
#neuromodulation
#retinitis pigmentosa
#gene therapy
#vision-restoration
#clinical trial
|
|
14
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|
SMCHD1 is a target for gene activation therapy to treat Prader-Willi syndrome.
(science.org)
|
Iminitoff M
…
Blewitt ME
Science Advances
2026-10-09
|
#epigenetics
#imprinting
#prader-willi
#gene therapy
#neurodevelopmental
|
|
9
|
|
Genome editing for hereditary angioedema: A landmark trial and important unanswered questions.
(linkinghub.elsevier.com)
|
Cancian M
…
Margaglione M
Cell Med
2026-10-09
|
#crispr
#gene therapy
#angioedema
#genome editing
#clinical trial
|
|
9
|
|
Use of split intein and myotropic AAV vectors enables effective preclinical gene therapy for dysferlinopathy.
(science.org)
|
McCormack NM
…
Tasfaout H
Science Translational Medicine
2026-10-07
|
#gene therapy
#muscular dystrophy
#aav vectors
#protein design
#muscle-disease
|
|
7
|
|
A translational pipeline for classification of variants of uncertain significance to support gene therapy clinical trial in OTOF-related deafness.
(linkinghub.elsevier.com)
|
Chen L
…
Shu Y
Cell Reports Medicine
2026-10-07
|
#gene therapy
#deafness
#variants
#congenital
|
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2
|
|
Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells.
(linkinghub.elsevier.com)
|
Luozhong S
…
Li B
Cell Stem Cell
2026-10-01
|
#genome editing
#hematopoietic
#stem-cells
#gene therapy
#nanoparticles
|
|
2
|
|
Silencing the mutant allele without compromising gene dosage.
(linkinghub.elsevier.com)
|
Tian L
…
Li Y
Cell Stem Cell
2026-10-01
|
#epigenetics
#gene therapy
#allele
#dominant-disorders
|
|
1
|
|
Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapy.
(nature.com)
|
Elia M
…
Lako M
Nature Communications
2026-09-30
|
#splicing
#gene therapy
#retinitis pigmentosa
#organoid
#spliceosome
|
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1
|
|
Integrating clinical diagnostics with interventional genetics for neurodevelopmental disorders.
(linkinghub.elsevier.com)
|
Macintosh J
…
Ernst C
American Journal of Human Genetics
2026-09-30
|
#neurodevelopmental
#sequencing
#gene therapy
#ipsc
#genetics
|