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Gene Therapy Volume 32 Issue 2
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Last updated over 1 year ago
Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
over 1 year ago
Retraction Note: Gene therapy with a promoter targeting both rods and cones...
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
over 1 year ago
A peptide conjugate enables systemic injection of the morpholino inducer and more...
AAV library screening identifies novel vector for efficient transduction of human aorta
over 1 year ago
AAV library screening identifies novel vector for efficient transduction of human aorta...
The circRNA circSCAF8 promotes tumor growth and metastasis of gastric cancer via miR-1293/TIMP1signaling
over 1 year ago
The circRNA circSCAF8 promotes tumor growth and metastasis of gastric cancer via...
CRISPR targeting of SNPs associated with age-related macular degeneration in ARPE-19 cells: a potential model for manipulating the complement system
over 1 year ago
CRISPR targeting of SNPs associated with age-related macular degeneration in ARPE-19 cells...
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
over 1 year ago
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact...
Chemogenetics with PSAM4-GlyR decreases excitability and epileptiform activity in epileptic hippocampus
over 1 year ago
Chemogenetics with PSAM4-GlyR decreases excitability and epileptiform activity in epileptic hippocampus Ana...
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration
over 1 year ago
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain...
Prime editing: therapeutic advances and mechanistic insights
over 1 year ago
Prime editing: therapeutic advances and mechanistic insights Joss B. MurrayPatrick T. HarrisonJanine...
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos
over 1 year ago
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos Tianyi ChengBaohui...
Unlocking patient access to gene therapy: five key practices
over 1 year ago
Unlocking patient access to gene therapy: five key practices Tay SalimullahBurcu Kazazoglu...