2022 Safadi Lecture: Emerging Gene Therapies for the Central Nervous System

Chicago, IL US
August 18, 2022
Adeno-associated viruses (AAVs) have been given to many patients with inherited diseases, with few drugs achieving regulatory approval because of issues with durability and toxicity. These issues generally arise because AAVs applied clinically show poor gene delivery efficiency to target tissues and cells of interest, requiring high doses. Also, gene expression cannot be regulated once introduced to cells. This session will discuss ongoing research that demonstrates progress in addressing these critical issues for the broader utility of AAVs for human therapies.

Learn more at our website!

Target Audience

This activity is designed for physicians and other healthcare professionals dedicated to the care of patients with inherited diseases.

Learning Objectives

After this activity, participants will be able to:

  • Describe current gene therapy tools to treat brain diseases;
  • Identify gain-of-function diseases, which are caused by the ectopic or increased activity of the mutated gene product;
  • Recall recent improvements in gene vectors and their design that will result in improved outcomes for patients with brain diseases.
Additional information
ACGME/ABMS Core Competencies: 
Medical Knowledge
For more information, please contact:
CME Coordinator Contact Name: 
Daniel Nava
CME Coordinator Contact Email: 
CME Coordinator Contact Phone: 
Available credit: 
  • 1.50 AMA PRA Category 1 Credit™
  • 1.50 Participation
Event starts: 
08/18/2022 - 11:30am
Event ends: 
08/18/2022 - 1:00pm
Activity opens: 
Activity expires: 
Knapp Center of Biomedical Discovery
900 E 57th St
Auditorium KCBD 103
Chicago, IL 60637
United States

Learners can attend this event in-person or virtually. For questions about the event, contact Daniel Nava via e-mail.

Learn more about the University of Chicago Department of Neurological Surgery by visiting their website!

ACCESSIBILITY The University of Chicago is committed to providing equal access appropriate to need and circumstances and complies fully with legal requirements of the Americans with Disabilities Act. If you are in need of special accommodation, please contact Amy Johnson via email at dnava@surgery.bsd.uchicago.edu.

The University of Chicago reserves the right to cancel or postpone this conference due to unforeseen circumstances. In the unlikely event this activity must be cancelled or postponed, the registration fee will be refunded; however, The University of Chicago is not responsible for any related costs, charges, or expenses to participants, including fees assessed by airline/travel/lodging agencies.

  Beverly L. Davidson, PhD
  Arthur V. Meigs Chair in Pediatrics
  Director, Raymond G Perelman Center for
  Cellular and Molecular Therapeutics
  Chief Scientific Strategy Officer
  Children’s Hospital of Philadelphia
  Professor, Pathology and Laboratory Medicine
                        University of Pennsylvania
Disclosure Declarations
As a provider accredited by the ACCME, The University of Chicago Pritzker School of Medicine asks everyone in a position to control the content of an education activity to disclose all financial relationships with any ineligible companies. This includes any entity whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients. Financial relationships are relevant if a financial relationship, in any amount, exists between the person in control of content and an ineligible company during the past 24 months, and the content of the education is related to the products of an ineligible company with whom the person has a financial relationship. Mechanisms are in place to identify and mitigate any relevant financial relationships prior to the start of the activity.

Additionally, The University of Chicago Pritzker School of Medicine requires Authors to identify investigational products or off-label uses of products regulated by the US Food and Drug Administration at first mention and where appropriate in the content.
Physician Credit
The University of Chicago Pritzker School of Medicine is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.
The University of Chicago Pritzker School of Medicine designates this live activity for a maximum of 1 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Other Healthcare Professional Credit
Other healthcare professionals will receive a Certificate of Participation. For information on the applicability and acceptance of Certificates of Participation for educational activities certified for AMA PRA Category 1 Credit™ from organizations accredited by the ACCME, please consult your professional licensing board.
Please Note: The credit claiming process will close three months after the conference end date. Requests to claim credit after three months will be subject to additional fees.
Registration: Click below to register for free!

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Please Note: The credit claiming process will close three months after the conference end date. Requests to claim credit after three months will be subject to additional fees.
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