Compositions for increasing survival of motor neuron protein (smn) levels in target cells and methods of use thereof for the treatment of spinal muscular atrophy
AssigneeRutgers, The State University of New Jersey
InventorsMegerditch KILEDJIAN, Mi ZHOU
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Abstract
Compositions for altering splicing activity of the DcpS gene and increasing SMN protein expression in target cells are provided. Also disclosed are methods of use of such compositions for the treatment of SMA.
Claims19
A method for screening agents which increase production of a DcpS variant in a cell for increasing production of survivor motor neuron (SMN) protein in spinal muscular atrophy (SMA) cells, comprising
- a) providing cells expressing the DcpS gene;
- b) incubating said cells in the presence and absence of an agent that modifies DcpS gene splicing pattern in order to increase production of a DcpSIn15 variant;
- and
- d) identifying agents which increase production of said DcpSIn15 variant, said increase in said variant causing an increase in survivor motor neuron (SMN) protein relative to untreated control cells.
A method for treatment or management of SMA in a patient in need thereof, comprising administration of an effective amount of an agent which increases survival motor neuron protein (SMN) levels in cells from said patient, thereby alleviating or modulating SMA symptoms in said patient.
Dependencies are inferred from the claim text.