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24
|
|
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
|
|
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
|
|
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
|
|
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
|
|
0
|
|
Etuvetidigene Autotemcel for the Treatment of Wiskott-Aldrich Syndrome.
(nejm.org)
|
Ferrua F
…
Aiuti A
New England Journal of Medicine
2026-09-24
|
#gene therapy
#hematopoietic stem cells
#immunodeficiency
#wiskott-aldrich-syndrome
#lentiviral vector
|
|
0
|
|
Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.
(linkinghub.elsevier.com)
|
Klatt D
…
Brendel C
Cell Stem Cell
2026-09-24
|
#gene therapy
#lentiviral
#sickle cell disease
#hematopoietic
#stem cell
|
|
0
|
|
Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.
(nature.com)
|
Xue F
…
Zhang L
Nature Medicine
2026-09-16
|
#gene therapy
#hemophilia
#aav
#coagulation
|
|
0
|
|
Structure-guided discovery and engineering of miniature CRISPR-Cas12m for epigenome editing.
(nature.com)
|
Yu T
…
Li K
Nature Structural & Molecular Biology
2026-09-16
|
#crispr
#epigenomics
#protein design
#structural biology
#gene therapy
|
|
0
|
|
Genetic principles enabling the creation of bacteria-based therapeutics.
(academic.oup.com)
|
Williams AB
…
Linke L
Genetics
2026-09-17
|
#therapeutics
#genetic engineering
#synthetic biology
#biocontainment
#gene therapy
|
|
0
|
|
Construction of a self-amplifying mRNA-LNP complex for phenylalanine hydroxylase and its efficacy evaluation in gene therapy for phenylketonuria.
(linkinghub.elsevier.com)
|
Wen Z
…
Fei J
iScience
2026-09-15
|
#rna
#gene therapy
#phenylketonuria
#lipid nanoparticles
#metabolic-disorders
|