Abstract
What is this summary about?
This is a summary of a review article. The article was about how long the effects of one type of gene therapy may last. Review articles summarize many published scientific articles. The original review article was published in the journal Molecular Therapy in March 2022.
Genes are inside cells and carry the information that determines a person's genetic makeup and traits (for example, their features or characteristics) that they get (inherit) from their parents. People living with genetic conditions have genes that do not work (function) like they should. Gene therapies have been developed for people living with serious rare health conditions. They aim to treat the cause of the condition instead of just managing symptoms. Gene therapy is designed to add a functioning copy of the gene into a person's cells. The functioning copy of the gene is inserted into cells by vectors.
Vectors are designed to act as vehicles to deliver the functioning copy of the gene into a person's cells. Often, these vectors are based on a virus because viruses are good at getting into the body. Vectors use the outer shell of the virus, but the viral genes are replaced with a therapeutic gene. Some vectors are modeled on the adeno-associated virus (AAV). This article is about AAV-based gene therapy and how long its treatment effects can last (durability or duration of treatment effect).
What conclusions did the authors make?
Further research is needed to understand what can affect how long the benefits of gene therapy will last.
Currently, AAV-based gene therapy can only be given once. An important aspect of successful gene therapy is its durability.
Research is ongoing to find ways to increase the durability of gene therapy.
Plain Language Summary
This is a plain language summary of a review article that was about how long the effects of gene therapy based on adeno-associated virus may last. There are many immunological and non-immunological factors that can affect how long gene therapy effects will last.
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This is an abstract of the Plain Language Summary of Publication article.
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Link to original article here
Financial disclosure
Disclosure information provided by the authors of the original review is available in the full text of the original article. Tara Moroz, Daniel Levy, and Martin Schulz are employees of and own stock in Pfizer. Laurence Woollard is an employee and sole shareholder of On The Pulse Consultancy Ltd, has received speaker/advisory committee honoraria from Novo Nordisk, Pfizer, and Roche, and received an honorarium from Pfizer as a patient author for this publication. Manish Muhuri and Guangping Gao have no conflicts to disclose. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Writing disclosure
Writing support for this summary was provided by Margit Rezabek, DVM, PhD, of Envision Pharma Group, Inc. and was funded by Pfizer.
Acknowledgments
The authors of the original article discussed in this summary are M Muhuri, DI Levy, M Schulz, D McCarty, and G Gao. The authors thank Stephanie Christopher and Lisa Wilcox for reviewing an earlier version of this plain language summary.
Competing interests disclosure
Tara Moroz, Daniel Levy, and Martin Schulz are employees of and own stock in Pfizer. Laurence Woollard is an employee and sole shareholder of On The Pulse Consultancy Ltd, has received speaker/advisory committee honoraria from Novo Nordisk, Pfizer, and Roche, and received an honorarium from Pfizer as a patient author for this publication. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.