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» QR-1011 restores ABCA4 deficiency in Stargardt disease
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Ocular
QR-1011 restores ABCA4 deficiency in Stargardt disease
Jan. 15, 2024
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Investigators from Proqr Therapeutics NV have tested their product QR-1011, an antisense oligonucleotide designed to correct splicing abnormalities within the
ABCA4
gene that affect protein expression levels, in Stargardt disease.
BioWorld Science
Genetic/congenital
Ocular