(Press-News.org) New Rochelle, NY, November 13, 2023—A new review article in the peer-reviewed journal Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases. The article also describes the challenges in the progress of gene therapy for rare diseases. Click here to read the article now.
Juan Bueren, from Centro de Investigaciones Energéticas Medioambientalies y Tecnológicas (CIEMAT), and President of the European Society for Gene and Cell Therapy (ESGCT), and Alberto Auricchio, from Telethon Institute of Genetics and Medicine (TIGEM), and Vice-President of the European Society for Gene and Cell Therapy, coauthored the article titled “Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases.”
“Advances in the generation of integration competent vectors have markedly improved the efficacy and also the safety associated to ex vivo hematopoietic stem cell gene therapies during the last decade,” state the authors. “In addition to ex vivo gene therapy, in vivo gene therapy has shown to be safe and effective therapy in humans. However, also some challenges remain to make this transformative therapeutic approach widely available.”
“Professors Bueren and Auricchio, as the leaders of the ESGCT, provide a uniquely comprehensive perspective on the development of molecular therapies for rare diseases in Europe,” says Editor in Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School.
About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.
About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.
END
Advances and challenges in gene therapy for rare diseases
2023-11-14
[Attachments] See images for this press release:
ELSE PRESS RELEASES FROM THIS DATE:
What factors influence PrEP prescribing behavior in health care providers?
2023-11-14
HIV pre-exposure prophylaxis (PrEP), a daily dose of two medications meant to prevent HIV infection in high-risk people, has changed public health dramatically in recent years. Yet, adolescents and young adults, one high-risk group, have shown slower uptake in using this prevention method.
Despite accounting for around 20 percent of new HIV infections, adolescents and young adults between the ages of 13 and 24 are still largely not being prescribed PrEP. Research has described physician intentions to prescribe PrEP to at-risk young people, but no studies until now have focused on factors that may affect actual prescribing of this evidence-based ...
ASCE establishes Dan M. Frangopol Medal for Life-Cycle Civil Engineering of Civil Structures
2023-11-14
The American Society of Civil Engineers (ASCE) recently instituted the Dan M. Frangopol Medal for Life-Cycle Engineering of Civil Structures in recognition of the Lehigh Engineering professor’s contributions as a pioneering researcher and educator and leading authority in the fields of life-cycle civil engineering and life-cycle cost optimization.
The award pays tribute to Frangopol, the inaugural Fazlur R. Khan Endowed Chair of Structural Engineering and Architecture in the Department of Civil and Environmental Engineering ...
Webb Telescope’s Marcia Rieke awarded Catherine Wolfe Bruce Gold Medal
2023-11-14
Dr. Marcia Rieke, principal investigator for the Near-Infrared Camera on NASA’s James Webb Space Telescope is the Astronomical Society of the Pacific’s (ASP) 2023 recipient of its most prestigious award. ASP’s Catherine Wolfe Bruce Gold Medal honors Rieke, a Regents Professor of astronomy and Elizabeth Roemer Endowed Chair, Steward Observatory, at the University of Arizona. Rieke’s award and achievements was recognized at the ASP Awards Gala on Saturday, Nov. 11, in Redwood City, California.
Groundbreaking Contributions
Rieke’s research has focused on infrared observations of ...
Galactic ‘lightsabers’: Answering longstanding questions about jets from black holes
2023-11-14
The one thing everyone knows about black holes is that absolutely everything nearby gets sucked into them.
Almost everything, it turns out.
“Even though black holes are defined as objects from which nothing can escape, one of the astonishing predictions of Einstein’s theory of relativity is that black holes can actually lose energy,” says astrophysicist Eliot Quataert, Princeton’s Charles A. Young Professor of Astronomy on the Class of 1897 Foundation. “They ...
Researchers identify unexpected twist while developing new polymer-based semiconductors
2023-11-14
CHAMPAIGN, Ill. — A new study led by chemists at the University of Illinois Urbana-Champaign brings fresh insight into the development of semiconductor materials that can do things their traditional silicon counterparts cannot – harness the power of chirality, a non-superimposable mirror image.
Chirality is one of nature’s strategies used to build complexity into structures, with the DNA double helix perhaps being the most recognized example – two molecule chains connected by a molecular “backbone” ...
Immigrants living in the U.S. have fewer preterm births
2023-11-14
Preterm birth predicts lifelong health outcomes
Worsening preterm birth rates in the U.S. represent a ‘key metric to target to improve overall societal health’
Study identifies key differences among Asian and Hispanic subgroups
Minority stress could contribute to inequities that begin at birth between populations in the U.S.
CHICAGO --- Preterm birth rates are an important marker in assessing a country’s overall health. And the United States isn’t fairing very well.
Individuals born in the U.S. had an overall higher rate (9.7%) of giving birth prematurely compared to U.S. immigrants (9%), a new Northwestern Medicine ...
When we see what others do, our brain sees not what we see, but what we expect
2023-11-14
When we see what others do, our brain sees not what we see, but what we expect
When we engage in social interactions, like shaking hands or having a conversation, our observation of other people’s actions is crucial. But what exactly happens in our brain during this process: how do the different brain regions talk to each other? Researchers at the Netherlands Institute for Neuroscience provide an intriguing answer: our perception of what others do depends more on what we expect to happen than previously believed.
For some time, researchers have been trying to understand how our brains process other people’s ...
Great results with emergency care adapted for pregnant women
2023-11-14
Increased vigilance for high blood pressure and diffuse stomach pain. These are some of the characteristics of emergency care adapted for pregnant women and new mothers. The model, which could become clinical routine throughout Sweden, is described in a thesis at the University of Gothenburg.
The aim of the thesis was to reduce morbidity and mortality among pregnant women and new mothers seeking emergency care. Sweden has relatively low rates of pregnancy-related morbidity and mortality, but pregnant women and new mothers do not currently receive ...
Vegan diet fosters changes in gut microbiome that reduce hot flashes by 95%, finds new study
2023-11-14
A low-fat vegan diet that includes soy fosters changes in the gut microbiome that decrease postmenopausal vasomotor symptoms, or hot flashes, overall by 95%, according to a new study by the Physicians Committee for Responsible Medicine. A vegan diet also eliminated severe hot flashes, led to a 96% decrease in moderate-to-severe hot flashes, and reduced daytime and nighttime hot flashes by 96% and 94%, respectively. Participants also lost 6.4 pounds on average.
“Women who want to fight hot flashes should feed the bacteria in their gut a vegan diet rich in fruits, vegetables, grains, and beans, which also leads to weight loss and protects against heart disease and type 2 diabetes,” ...
Drug that kills off sleeping bone cells could treat lower back pain
2023-11-14
An existing drug that targets senescent, or sleeping cells could provide the answer to treating lower back pain, according to a new study.
The research, published today as a Reviewed Preprint in eLife, advances our understanding of the role of senescent osteoclasts – cells that break down and remove damaged bone tissue – in the development of lower back pain, which affects 8 in 10 people at some point in their lives. eLife’s editors say the study provides compelling evidence that an existing drug, Navitoclax, can eliminate senescent osteoclasts in mice and, in doing so, markedly reduce spinal pain.
Osteoclasts resorb ...