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 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.
You can learn more about how clinical trials work here and here. To search for clinical trials that are relevant to you/your loved one, please visit: https://clinicaltrials.gov/ To access a curated list of gene and cell therapy trials (sourced from clinicaltrials.gov) you can visit the ASGCT Clinical Trials Finder. Talk to your health care provider before enrolling in a clinical trial as they can help you assess the legitimacy of the trial.
How long before gene editing therapies will become available for my/my loved one’s genetic disease?
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.
I’d like to learn more about gene editing; where can I access educational materials?
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?
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)
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 (email@example.com) with any follow-up questions you may have.
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