Gene Transfer, Editing, Silencing and RNA Based‐Approaches: What, When and How?

Released On
April 22, 2021

Expires On
April 29, 2022

Media Type

Completion Time
60 minutes

Hepatology, Neurology, Primary Care, Endocrinology, Pulmonology, Cardiology

Gene Therapy

Scroll to the Bottom of this Information to Begin this Course

This activity is jointly provided by by Postgraduate Institute for Medicine (PIM) and PlatformQ Health Education, LLC working in collaboration with the National Organization for Rare Disorders (NORD).


Supported by independent educational grants from Astellas Gene Therapies; AVROBIO, Inc.; Biogen; CRISPR Therapeutics, Inc.; Helixmith Co., Ltd; Orchard Therapeutics; REGENXBIO Inc.; and uniQure, Inc.

Credit Available

  • Physicians — maximum of 1.0 AMA PRA Category 1 Credit(s)
  • Physician Assistants — maximum of 1.0 AAPA Category 1 CME credits
  • Pharmacists — 1.0 Contact Hours (0.01 CEUs)
  • Nurses — 1.0 Contact Hours
  • Pshychologists — 1.0 Continuing Education Credit
  • Social Workers — 1.0 Clinical Continuing Education Credits

All other healthcare professionals completing this course will be issued a statement of participation.

Target Audience

This activity is intended for primary care providers, internal-medicine specialists, pediatricians, pulmonologists, cardiologists, endocrinologists, gastroenterologists, hepatologists, neurologists, neuromuscular specialists, infectious-disease specialists, obstetricians/gynecologists, rheumatologists, dermatologists, hematologists, oncologists, neonatologists, genetic counselors, social workers, psychologists, nurse practitioners, physician assistants, nurses, and pharmacists

Learning Objectives

Upon completion of this activity, participants should be better able to:

  • Explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions
  • Discuss different types of current and emerging gene‐based therapies


Alessandra Biffi, MD

Professor of Pediatrics
Director, Division of Pediatric Hematology,
Oncology and Stem Cell Transplant
University of Padua

Kevin Flanigan, MD

Director, Center for Gene Therapy
Robert F. & Edgar T. Wolfe Foundation
Endowed Chair in Neuromuscular Research
Director, Nationwide Children’s Hospital
Center for Research Translation in Muscular Dystrophy
Therapeutic Development
Abigail Wexner Research Institute Nationwide Children’s Hospital
Professor of Pediatrics and Neurology
The Ohio State University

Kimberly Goodspeed, MD

Assistant Professor of Pediatrics,
Neurology Division
University of Texas Southwestern Medical
Center Dallas

Stephan A. Grupp, MD, PhD

Section Chief, Cellular Therapy and Transplant
Director, Cancer Immunotherapy Program
Children’s Hospital of Philadelphia
Novotny Professor of Pediatrics
Perelman School of Medicine at the
University of Pennsylvania

John Kessler, MD

Davee Professor
Davee Department of Neurology
Northwestern University

Julie A. Parsons, MD

Haberfeld Family Endowed Chair in
Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine
Children's Hospital Colorado

Perry B. Shieh, MD, PhD, FAAN

Professor of Neurology and Pediatrics
David Geffen School of Medicine at UCLA
University of California, Los Angeles

Joint Accreditation Statement

In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and PlatformQ Health Education, LLC. Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Credit Designation Statements

Physician Continuing Medical Education

The Postgraduate Institute for Medicine designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

Please note, MOC credit is reported on a monthly basis.

To claim and receive MOC credits, learners must provide:

  • ABIM ID number
  • First and last name
  • Degree and specialty
  • DOB (MMDD)
  • Activity completion date

Nursing Continuing Education

The maximum number of hours awarded for this Continuing Nursing Education activity is 1.0 contact hours.

Physician Assistant Education

Postgraduate Institute for Medicine has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 1.0 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.

Pharmacist Continuing Education

Postgraduate Institute for Medicine is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. Postgraduate Institute for Medicine designates this continuing education activity for 1 contact hours (0.01 CEUs) of the Accreditation Council for Pharmacy Education.

(Universal Activity Number - JA4008162-9999-21-2661-H01-P)

Type of Activity: Application

Pharmacists will need to provide their National Association of Boards of Pharmacy (NABP) e-Profile ID and date of birth (MMDD) to ensure the data is submitted to the ACPE and NABP properly. If you need to obtain your NABP e-Profile, please visit

APA Education (American Psychology Association)

Continuing Education (CE) credits for psychologists are provided through the co-sponsorship of the American Psychological Association (APA) Office of Continuing Education in Psychology (CEP). The APA CEP Office maintains responsibility for the content of the programs. This program offers 1.0 continuing education credit for psychologists.

Social Work Education

As a Jointly Accredited Organization, Postgraduate Institute for Medicine is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved under this program. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit. Postgraduate Institute for Medicine maintains responsibility for this course. Social workers completing this course receive 1.0 clinical continuing education credits.

Disclosures of Conflicts of Interest

Postgraduate Institute for Medicine (PIM) requires instructors, planners, managers and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy.

PIM is committed to providing its learners with high quality CME activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Alessandra Biffi, MD

  • Receipt of Intellectual Property Rights/Patent Holder: Altheia Science, Orchard Therapeutics
  • Consulting Fee: Altheia Science, Orchard Therapeutics
  • Contracted Research: Altheia Science, Orchard Therapeutics
  • Ownership Interest less than 5%: Orchard Therapeutics
  • Ownership Interest greater than 5%: Altheia Science

Kevin Flanigan, MD

  • Royalty: Audentes
  • Consulting Fees: Apic Bio, 4D Molecular Theraputics
  • Contracted Research: Audentes
  • Ownership Interest less than 5%: 4D Molecular Therapeutics

Kimberly Goodspeed, MD

  • Salary: Taysha Gene Therapies - Support for work on gene therapy translational research program

Stephan A. Grupp, MD, PhD

  • Consulting Fees: Adaptimmune, Allogene, CBMG, Cabaletta, Cellectis, GSK, Humanigen, Janssen/JnJ, Jazz, Juno, Novartis, Roche, TCR2, Vertex
  • Contracted Research: Kite, Novartis, Servier, Vertex

John Kessler, MD

  • Contracted Research: Support of clinical trials at 26 institutions including Northwestern. No funds to my lab or to me personally. I am the national PI of the trials but not the site PI at Northwestern.

Julie A. Parsons, MD

  • Consulting Fees: Biogen, Genentech, Novartis, Scholar Rock
  • Contracted Research: Biogen, Novartis, Scholar Rock

Perry B. Shieh, MD, PhD, FAAN

  • Consulting Fees: Alexion, Biogen, Genentech, Pfizer, PTC, Sarepta
  • Fees for Non-CE Services Received Directly from an Ineligible Entity or their Agents: Alexion, Avexis, Biogen, CSL Behring, Genentech, Grifols
  • Contracted Research: Avexis, Biogen, Pfizer, PTC, Sarepta

The planners and managers reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Carole Drexel, Medical Director PlatformQ Health Education, LLC: None.

The PIM planners and managers have nothing to disclose.

The following PlatformQ Health Education, LLC planner, Alissa Yost, hereby states that she or her spouse/life partner do not have any financial relationships or relationships to products or devices with any commercial interest related to the content of this activity of any amount during the past 12 months.

Disclosure of Unapproved/Off Label Use (if applicable)

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

Method of Participation

There are no fees to participate in this activity. To participate in the activity, go to To receive credit, participants must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a minimum score of 70% on the post-test.

Hardware/Software Requirements

Participants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.


Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Contact Information

Accreditation Support:
Please contact the Postgraduate Institute for Medicine at

Technical Support:
For any technical issues or issues with your CME Certificate, please contact ClinicalSeriesLive at 877-394-1306 or at

PlatformQ Health Privacy Policy:

By clicking “Start Activity” you are agreeing to PlatformQ Health’s privacy policy. PlatformQ Health takes your privacy very seriously. The full Privacy Policy found here describes (i) the types of information we collect from you when you visit our Site; (ii) how we use the information we collect; (iii) with whom and why we share it; and (iv) the choices we offer regarding our use of the information. We also describe how to contact us about our privacy practices.

Information we collect is primarily used to communicate with program participants, award CME/CE credits, manage user accounts, recommend relevant programs and perform data analysis to inform educational outcomes.

In plain English, here is what we affirm:

  • You can change your registration profile at any time from our “Profile” page
  • To request any of these changes without logging in, please call us at 1 (877) 394-1306 or email us at
  • You can end your association with PlatformQ Health at any time
  • PlatformQ Health does not spam, nor we do we rent or sell our email list in whole or in part. Any program-related data sharing is for engagement verification and opt-in based further education
  • EU citizens can request a complete record of their account and/or request to have their personal data expunged by contacting with the subject line “Data Request”

You acknowledge and agree that by beginning this activity, you have affirmatively agreed to this Privacy Policy and consented to receiving electronic messages in conformance with this Privacy Policy. Please read the full privacy policy located at: