Frequently Asked Questions 

Gene editing treatments for several genetic diseases such as sickle-cell anemia, T-cell leukemia, familial hypercholesterolemia, and transthyretin amyloidosis are already in clinical trials. To advance experimental gene editing therapeutics into clinical trials for other genetic diseases, preclinical data needs to convincingly demonstrate that patients would benefit from having their disease-associated gene(s) edited and that any predicted benefits would significantly outweigh any potential side effects.

Developing a therapeutic gene editing strategy that maximizes the therapeutic effect and minimizes the risk of unwanted side effects requires optimizing several parameters such as:

• Ideal timing of therapeutic intervention for maximal benefit. The consequence of administering a therapeutic gene editing strategy for a given disease often varies depending on the patient’s age, and the different stages of the disease. Determining the ideal time to intervene requires sufficient knowledge of disease progression.
• Determining the target tissues or cell types to edit. Ensuring that gene editors selectively target the cell and tissue types affected by the disease will help minimize unwanted “off-target” effects that might result from editing otherwise healthy cells and tissues.
• Safe and effective delivery methods that can route gene editing agents to the desired cells and tissues. In contrast to small molecules such as aspirin or ibuprofen, gene editors are large macromolecules that require creative delivery strategies that are more complex than swallowing a pill.
• Data from animal models of the disease that demonstrate that the candidate gene editing strategy is likely to benefit the patient without any evident unacceptable risk of side effects.
• A path to manufacture the candidate therapeutic at the quality standard required for a clinical trial.

Finally, regulatory approval is required to initiate the trial, which often requires the above information and additional data.

The Innovative Genomics Institute or IGI has developed educational materials for anyone interested in learning more about genome editing: https://innovativegenomics.org/education/

The American Society of Gene + Cell Therapy (ASGCT)’s Patient Education Program

A Glossary of Genomic/Genetic Terms by the National Human Genome Research Institute (NHGRI) https://www.genome.gov/genetics-glossary

A non-exhaustive list of resources is below:

• Global Genes
• Genetic and Rare Diseases (GARD) Information Center
• Genetic Alliance
• MyGene2
• Undiagnosed Diseases Network
• Rare Genomics Institute
• Rare Diseases Clinical Research Network
• National Organization for Rare Disorders (NORD)

There are a few ways to stay up-to-date on our group’s latest happenings! Aside from visiting our publications page to freely access and download our research papers you can: 

FOLLOW US on social media

• https://twitter.com/liugroup (our lab)
• https://twitter.com/davidrliu (Professor Liu)

LISTEN to one of Professor Liu’s public lectures (TED Talk; CNN’s Life Itself Lecture) or podcast interviews with Professor Liu (Theory & Practice; People Behind the Science Podcast; Bios Podcast; For Your Innovation Podcast)

READ some recent interviews with Professor Liu (The Conversation; GEN Edge; Endpoints News) or news stories written about our group’s work (press page)

Please contact the Liu Group Manager of Communications and Outreach: Anahita Vieira (Ph.D.) via email at (avieira@broadinstitute.org) with any follow-up questions you may have.