UBE3A for treating angel syndrome

A protein and polynucleotide technology, applied in the field of UBE3A for the treatment of Angelman syndrome

Pending Publication Date: 2022-05-13
RGT UNIV OF CALIFORNIA
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

There are no effective therapies available for the treatment of AS

Method used

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  • UBE3A for treating angel syndrome
  • UBE3A for treating angel syndrome
  • UBE3A for treating angel syndrome

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Embodiment approach

[0140] Polynucleotides and Peptides

[0141] The present disclosure provides polynucleotides encoding ubiquitin protein ligase E3A (Ube3a) proteins, polypeptides or biological equivalents thereof. In some embodiments, polynucleotides are recombinant and / or isolated. In some embodiments, the Ube3a protein, polypeptide, or biological equivalent thereof comprises one or more glycosylation sites.

[0142] Also provided are Ube3a proteins, polypeptides or biological equivalents thereof comprising one or more glycosylation sites. In some embodiments, the Ube3a protein, polypeptide or biological equivalent thereof is isolated, engineered and / or recombinant.

[0143] In some embodiments, the Ube3a protein, polypeptide or biological equivalent thereof is not a wild-type Ube3a protein, for example comprising a sequence selected from SEQ ID NO: 8, 10, 12, 20, 22 or 24 or any natural variant thereof . Such Ube3a proteins, polypeptides or biological equivalents thereof are also referre...

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Abstract

The angel syndrome is a hereditary neurological disorder, and is characterized by development retardation, intellectual disability, serious speech disorder and exercise and balance problems. Provided herein are polynucleotides, vectors, polypeptides, cells, compositions, kits and methods for treating angel syndrome.

Description

[0001] Cross References to Related Applications [0002] This application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Application Nos. 62 / 890,364 and 62 / 945,062, filed August 22, 2019, and December 6, 2020, respectively, the contents of each of which are adopted by References are incorporated in this application in their entirety. Background technique [0003] Angelman syndrome (AS) is an inherited neurological disorder characterized by developmental delay, intellectual disability, severe speech impairment, and problems with movement and balance. Most patients have recurrent seizures and small head size. Most patients show developmental delay, and other common symptoms appear in early childhood. Children with AS often have a happy and excited demeanor. Other symptoms include hyperactivity, short attention span and fascination with water. People with Angelman syndrome become less aroused and sleep problems improve as patients age. However, these patients wi...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K38/43A61P25/00C12N15/63C12N15/52
CPCA61P25/00C12N15/86C12N2740/16043C12N9/104A61K38/00C12Y203/02A61P25/28A61K38/53C12N5/0647C12N2740/15043
Inventor 约瑟夫·安德森
Owner RGT UNIV OF CALIFORNIA
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