Antisense oligonucleotides for the treatment of Leber congenital amaurosis
AssigneeStichting Katholieke Universiteit
InventorsRobert Wilhelmus Johanna Collin, Franciscus Peter Maria Cremers, Antonia Ingrid Den Hollander
Life of this patent
Estimating the term…
Abstract
The present invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Leber congenital amaurosis.
Claims14
An antisense oligonucleotide that is capable of modulating splicing of CEP290 c.2991+1655A>G, wherein the antisense oligonucleotide consists of 14 to 20 nucleotides that are 100% complementary to a sequence within SEQ ID NO:
- 6 and wherein the antisense oligonucleotide comprises one or more sugar moieties that are mono- or di-substituted at the 2′, 3′ and/or 5′ position.
A viral vector expressing an antisense oligonucleotide that is capable of modulating splicing of CEP290 c.2991+1655A>G, wherein the antisense oligonucleotide consists of 14 to 20 nucleotides that are 100% complementary to SEQ ID NO:
- 6.
Dependencies are inferred from the claim text.