Frequently Asked Questions 

Answers to some of the most common questions we receive are listed below as a resource for patients and their loved ones. Please feel free to email us (drliuresearch@broadinstitute.org) with any follow-up questions.

Please note that we cannot offer medical advice or interpret test results.

How can I sign up myself/my loved one for participation in gene editing clinical trials?

While our group is working intensely on maximizing the types of genetic diseases that could potentially benefit from gene editing technologies, we do not run any human clinical trials. As a research lab, we work on the preclinical side by developing and testing gene editing agents in cultured cells and in animal models of human genetic diseases. Our hope is that the resulting research will enable scientists in industry or other clinical organizations to develop safe and effective therapeutics for gene editing clinical trials.

To read more about clinical trials and how they work:

To search for clinical trials that are relevant to you/your loved one:

Please consult with your healthcare provider before enrolling in a clinical trial, as they can help you assess the legitimacy and appropriateness of the trial.

How long before gene editing therapies will become available for my/my loved one’s genetic disease?
Although we can’t predict exactly when gene editing will be available for a each rare disease, we are encouraged by the fact that at least 19 clinical trials using base editing or prime editing are currently underway to treat diseases, such as leukemias, hypercholesterolemia, alpha-1 antitrypsin deficiency, sickle-cell disease, beta-thalassemia, and chronic granulomatous disease. While there are still a few important scientific issues to resolve (such as the safest and most effective way to deliver these gene editors), most of the barriers we face today are either financial or regulatory in nature. We therefore encourage rare disease patients and advocates to contact their representatives and ask them if there are ways to encourage the FDA to streamline the process of translating gene editing from bench to bedside.
 
What scientific challenges remain?

Developing a therapeutic gene editing strategy that maximizes the therapeutic effect and minimizes the risk of unwanted side effects will also require optimizing the following parameters: 

  • 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.
I’d like to learn more about gene editing; where can I access educational materials?

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

Where can I learn more about rare diseases and/or connect with other patients and patient advocates?

A non-exhaustive list of resources is below:

How can I keep up with the latest news from the Liu group?

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

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)

Who should I contact if I have follow-up questions?

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. 

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