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gene therapy

28th Nov 2025

Cystic fibrosis plain language summary: exploring gene therapy possibilities

30th Jul 2025

Plain language summary: 4-year outcomes from testing valoctocogene roxaparvovec gene therapy in people with hemophilia A

27th May 2025

Plain language summary: Five-year results of nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer

21st Mar 2025

A plain language summary of the two-year results from a five-year study on giroctocogene fitelparvovec gene therapy for individuals with severe hemophilia A

17th Mar 2025

A plain language summary comparing bleeding outcomes after valoctocogene roxaparvovec gene therapy versus emicizumab prophylaxis

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
30th Apr 2024

A plain language summary looking at how the effects of gene therapy based on adeno-associated virus last

eople diagnosed with a disease called large B-cell lymphoma (LBCL) may experience return, or early relapse, of their disease within the first year after receiving and responding to their first (first-line) treatment regimen. Others may have primary refractory disease, meaning that the disease either did not respond to first-line treatment at all or only responded for a very brief period. Second (second-line) treatment includes immunotherapy followed by high-dose chemotherapy and ASCT, which has the potential to cure LBCL. However, if the disease does not respond to immunotherapy, people cannot receive ASCT, and less than 30% of people are cured.Therefore, new second-line treatment options are required, such as CAR T cell therapy, which uses a person’s own genetically engineered lymphocytes, also called T cells, to fight their lymphoma. In this article, we summarize the key results of the phase 3 TRANSFORM clinical studythat tested if liso-cel, a CAR T cell treatment, can safely and effectively be used as a second-line treatment for people with early relapsed or primary refractory (relapsed/refractory) LBCL
30th Apr 2024

A plain language summary on the TRANSFORM study: liso-cel as a second treatment regimen for large B-cell lymphoma

his is a summary of a review article about gene therapy. Review articles summarize many previously published scientific articles. The review was about how a common virus could change how one type of gene therapy works. It also discussed the importance of antibody testing for people who may receive gene therapy. The original review was published in Molecular Therapy in 2023
22nd Jan 2024

A plain language summary of the challenges and solutions for people with antibodies against AAV-based gene therapy

In healthy people, a protein called factor VIII (FVIII) helps blood to clot and prevents excessive bleeding. People with hemophilia A lack FVIII because a faulty F8 gene is giving the wrong instructions to the liver cells that make it. Valoctocogene roxaparvovec (ROCTAVIAN™) is a gene therapy designed to transfer working copies of the F8 gene into liver cells. This summary describes the GENEr8-1 study, which looked at how well valoctocogene roxaparvovec works for treating people with severe hemophilia A compared with their usual FVIII replacement therapy, and its safety. 134 men received valoctocogene roxaparvovec; results from the first 2 years are reported.
14th Nov 2023

Plain Language Summary of Publication article: the GENEr8-1 study of valoctocogene roxaparvovec gene therapy for hemophilia A

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