Cystic fibrosis (CF) is a life-shortening genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Over the past decade, the U.S. Food and Drug Administration (FDA) has approved four mutation-targeted therapies that enhance the function of the CFTR protein. These therapies have significantly improved the overall quality of life for many people with cystic fibrosis. However, it is essential to note that roughly 10% of people with CF do not benefit from these approved modulator therapies—whether due to ineligible genetic mutations, side effects, or suboptimal response—and continue to face a substantial disease burden, which affects both their physical and mental health. Gene therapy shows great promise as a potential cure for cystic fibrosis. However, past challenges in this field underline the critical importance of ensuring safety in developing gene therapies. Additionally, safety is a significant concern for individuals with CF participating in clinical trials, as highlighted by the "Final 10% Survey" conducted by Emily's Entourage in 2021 and 2022. Negative results are a crucial source of information regarding the safety and effectiveness of gene therapies. Negative results are findings that do not show statistical significance or fail to confirm the hypothesis despite thorough experimentation. The failure to publish negative results leads to research teams repeating studies that have already shown no effect or side effects, costing time and resources that could have been allocated to a different treatment. Moreover, the lack of published negative results impacts patients who do not see clinical trials demonstrating adverse events published in peer-reviewed journals. Sharing these results is vital to prevent harm to future patients participating in a duplicate trial. For this Special Issue, we encourage researchers to submit brief communications reporting negative results from early-stage, pre-clinical, and clinical gene therapy studies focused on CF. By highlighting these findings, we can foster transparency in research and advance gene therapy for cystic fibrosis. References: 1. Emily’s Entourage. The Final 10% Survey Report [Internet]. [cited 2024 Aug 3]. Available from: https://www.emilysentourage.org/wp-content/uploads/ee-final-10-percent-survey.pdf 2. Kramer-Golinkoff E, Camacho A, Kramer L, Taylor-Cousar JL. A survey: Understanding the health and perspectives of people with CF not benefiting from CFTR modulators. Pediatr Pulmonol. 2022;57(5).

See more information on the new Gene Therapy collection on genetic therapies for eye and brain.

The Editor-in-Chief of Gene Therapy is delighted to share with you an Editor's Choice selection of key papers that highlight some of the best current research published in the journal. These papers showcase the breadth of scope and coverage that Gene Therapy consistently deliver to its readers, and have been selected because readers of the journal will find them particularly interesting or useful.

Genome Engineering has great potential to change how we model, understand, and treat diseases.

In this collection we highlight a selection of articles from 2021, which top the list of the journal’s most cited, downloaded and most shared (including press coverage, blogs, Twitter, Facebook and Weibo). They showcase the breadth of scope and coverage that the journal consistently delivers to its readers.

In less than 10 years since the seminal paper by Doudna and Charpentier, the discovery of CRISPR as a programmable gene editing tool has revolutionized the therapeutic genome editing field. This was acknowledged last year with the announcement of the Nobel prize in Chemistry and continues to be exemplified by increasing publications on the subject. In the last two years, Gene Therapy has seen a remarkable increase in gene editing papers ranging from the basics, such as the contributions of mitotic gene conversion through to in vivo editing of Tay Sachs and Sandoff disease. In this collection, we have selected the most recent papers making a contribution to this exciting field.

See what scientists worldwide have been reading, citing and sharing.

One of the most exciting aspects emerging from the field of gene therapy is that it demonstrates how it can be applied to targeting cancer cells. As cancer is rarely a monogenic disease, this demonstrates the extraordinary reach that gene therapy applications have beyond their original use in addressing those orphan diseases many of us have worked on during our careers. The papers in this collection use standard AAV based delivery approaches, apply AI algorithms, and argue the pros and cons of genome engineering towards CAR-T treatments. This collection is a validation that basic science can be used to build on a greater foundation of scientific capital which can be applied to many aspects of human health.

The Editors of Eye and Gene Therapy have put together this joint collection on gene therapy in the eye, to showcase some of the recent research in this field.

Research in regenerative medicine is thriving across the world, and América del Sur is no exception. This special issue of Gene Therapy, coordinated by South America Associate Editor Ursula da Silveira Matte and Guest Editor Hugo Peluffo Zavala, is devoted to work submitted by authors located in many South American countries, with topics including cancer, cardiovascular, Parkinson and inherited diseases. The papers report use of multiple gene and cell therapy technologies, spanning viral and non-viral vectors, genome editing, DNA vaccines, chimeric antigen receptor T-cells (CAR-T), induced pluripotent stem cells (iPSC), and adipose-tissue-derived stem cells.

See what scientists world-wide have been citing, reading and sharing. In this Web Collection we highlight a selection of articles from 2018 and 2019 which top the list of the journal's most cited, most read and most shared (including press coverage and Twitter). They showcase the breadth of scope and coverage that Gene Therapy consistently delivers to its readers.

To celebrate 25 years of Gene Therapy, the Editor-in-Chief has selected 25 of the journal's most innovative papers to highlight the journal's impact in the field over the past 25 years.