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Current Pipeline
CRISPR

UNC

University of North Carolina (UNC) is exploring an investigational viral delivered Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CAS9 approach to treating Angelman syndrome.

Pre-clinical

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

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Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

A gene editing tool that utilizes a guide RNA (gRNA) to recognize a specific section of DNA or RNA and directs an enzyme (nuclease) to cut at a specific section in the DNA or RNA. Designing a CRISPR that affects the UBE3A-ATS could potentially allow the paternal UBE3A gene to be turned on. It could also be used to edit a mutation in a gene, known as base editing.

Recent Updates

  • April 2023

    Bench to bedside, Carolina leads study of Angelman syndrome

    Read more
  • Oct 2020

    Scientists Take Major Step Toward Angelman Syndrome Gene Therapy

    Read More
  • Sept 2019

    Zylka Lab awarded $6.1 million from NIH to develop CRISPR/Cas9 gene therapy for Angelman syndrome, study UBE3A autism gene with Philpot Lab

    Read more

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.