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US 2020/0370069 A1Published application
Treatment of spinal muscular atrophy
AssigneesGenethon, Inserm (Institut National De La Sante Et De La Recherche Medicale), Universite D'Evry Val D'Essonne
InventorsANA BUJ BELLO, MARTINA MARINELLO, SAMIA MARTIN
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Abstract
The present invention relates to a recombinant adeno-associated virus (rAAV) vector comprising a serotype 9 or rh10 AAV capsid, for use in a method for the treatment of spinal muscular atrophy (SMA).
Claims21
1-45 (canceled)
A rAAV vector comprising:
- (a)(i) an AAV9 capsid or an AAVrh10 capsid;
- and
- (a)(ii) a single-stranded genome which is not self-complementary, said genome comprising a gene coding a human survival motor neuron (SMN) protein under the control of a CAG promoter;
- or
- (b)(i) a capsid selected from an AAV9 capsid and an AAVrh10 capsid;
- and
- (b)(ii) a single-stranded genome which is not self-complementary, said genome containing a nucleic acid sequence comprising, in this order:
- an AAV 5′-ITR;
- a CAG promoter;
- a gene coding a human survival of motor neuron (SMN) protein;
- optionally, a further regulatory element;
- a HBB2 polyadenylation signal;
- and
- an AAV 3′-ITR;
- wherein the further regulatory element is not a WPRE;
- or
- (c)(i) an AAV9 capsid or an AAVrh10 capsid;
- and
- (c)(ii) a single-stranded genome which is not a self-complementary genome including a gene coding a human survival motor neuron (SMN) protein, wherein said genome does not comprise a WPRE and wherein said genome does not comprise a SV40 intron.
16 more elements
An isolated nucleic acid sequence comprising, in this order:
- an AAV 5′-ITR, a promoter, a gene encoding a SMN protein, a polyadenylation signal and an AAV 3′-ITR, wherein said isolated nucleic acid is configured to form a single-stranded AAV genome which is not self-complementary.
Dependencies are inferred from the claim text.