Promising results reported in study of AAV-based gene delivery to treat methylmalonic acidemiaApril 05, 2016
New Rochelle, NY, April 1, 2016--A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated viral (AAV) vectors being developed for gene therapy to replace the enzyme (MUT) that is deficient in MMA. These results, and especially the absence of AAV antibodies in most patients who completely lack the MUT enzyme and have not had a liver transplant--those in greatest need of improved therapies--has encouraging implications for gene delivery as a treatment for MMA, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Human Gene Therapy website until May 1, 2016.
Elizabeth Harrington, Jennifer Sloan, Irini Manoli, Randy Chandler, Charles Venditti, and Peter McGuire, National Human Genome Research Institute, National Institutes of Health (Bethesda, MD), and Mark Schneider, Roberto Calcedo, and James Wilson, Perelman School of Medicine, University of Pennsylvania (Philadelphia), coauthored the article entitled "Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia (MMA)".
AAV is proving to be an effective gene transfer vehicle for systemic gene delivery. The authors have developed and tested AAV vectors that carry the mouse mut gene and have reported promising results of gene transfer in a mouse model of MMA. They have also developed an AAV vector to deliver the human MUT gene. The presence of pre-existing antibodies to AAV capsid proteins can trigger an immune reaction against the vector and interfere with delivery of the therapeutic gene.
"The presence of neutralizing antibodies in some patients is one of the few remaining obstacles to successful correction of genetic diseases with AAV vectors," 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, Worcester, MA. "The observation that anti-AAV antibody levels are lower than expected in MMA patients bodes well for the future success of AAV-based gene therapy with this disorder."
Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. 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, Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its companion journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.
About the Publisher
Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.
Mary Ann Liebert, Inc./Genetic Engineering News
Related Gene Therapy Articles:
Drugs that are used in cancer therapy to erase epigenetic alterations in cancer cells simultaneously promote the production of countless mysterious gene transcripts, scientists from the German Cancer Research Center now report in Nature Genetics.
A single treatment giving life-long protection from severe allergies such as asthma could be made possible by immunology research at The University of Queensland.
Twenty-five years have passed since the publication of the first work on solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) as a system for delivering drugs.
A team headed by Professor Albert Rizvanov, director of the Gene and Cell Technologies Open Lab, created a gene therapy drug that encodes growth factors for the stimulation of blood vessel and bone formation.
A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy.
A new gene therapy could be used to prevent the loss of muscle mass and physical strength associated with advanced cancer
The ability to switch disease-causing genes on and off remains a dream for many physicians, research scientists and patients.
A team from the International School for Advanced Studies (SISSA) in Trieste has obtained very promising results by applying gene therapy to glioblastoma.
Elizabeth Parrish, CEO of Bioviva USA Inc. has become the first human being to be successfully rejuvenated by gene therapy, after her own company's experimental therapies reversed 20 years of normal telomere shortening.
Scientists in the Barabas group at EMBL have increased the efficiency of a genome-engineering tool called Sleeping Beauty, which is showing promise in clinical trials for leukemia and lymphoma immunotherapies.
Related Gene Therapy Reading:
The Forever Fix: Gene Therapy and the Boy Who Saved It
by Ricki Lewis (Author)
Fascinating narrative science that explores the next frontier in medicine and genetics through the very personal prism of the children and families gene therapy has touched.
Eight-year-old Corey Haas was nearly blind from a hereditary disorder when his sight was restored through a delicate procedure that made medical history. Like something from a science fiction novel, doctors carefully introduced viruses bearing healing genes into Corey's eyes―a few days later, Corey could see, his sight restored by gene therapy.
THE FOREVER FIX is the first book to tell the fascinating story... View Details
The Gene: An Intimate History
by Siddhartha Mukherjee (Author)
THE #1 NEW YORK TIMES BESTSELLER
A New York Times Notable Book
A Washington Post and Seattle Times Best Book of the Year
From the Pulitzer Prize-winning author of The Emperor of All Maladies—a fascinating history of the gene and “a magisterial account of how human minds have laboriously, ingeniously picked apart what makes us tick” (Elle).
“Dr. Siddhartha Mukherjee dazzled readers with his Pulitzer Prize-winning The Emperor of All Maladies in 2010. That achievement was evidently just a warm-up for... View Details
The Gene Therapy Plan: Taking Control of Your Genetic Destiny with Diet and Lifestyle
by Mitchell L. Gaynor MD (Author), Mehmet C. Oz M.D. (Foreword)
Your genes are not your destiny: learn to prevent disease, improve brain function, and reverse the course of obesity and premature aging through easy-to-adopt nutrition and lifestyle changes that target your DNA
While we cannot alter the genes we are born with, we can prevent and reverse disease with foods, supplements, and lifestyle choices that turn good genes on and bad genes off. In his pathbreaking plan, Dr. Mitchell Gaynor—a renowned oncologist and pioneer in integrative medicine—focuses on obesity, heart disease, diabetes, cancer, and aging to explain... View Details
Gene Therapy in Neurological Disorders
by Mingjie Li (Editor), B. Joy Snider (Editor)
Gene therapy has tremendous potential for the treatment of neurological disorders. There has been substantial progress in the development of gene therapy strategies for neurological disorders over the last two decades. Gene Therapy in Neurological Disorders thoroughly reviews currently available gene therapy tools and presents examples of their application in a variety of neurological diseases. The book begins with general reviews of gene therapy strategies with a focus on neurological disorders. The remainder of the chapters present approaches to specific neurological disorders.... View Details
Gene Therapy in Reconstructive and Regenerative Surgery
by Giorgio Giatsidis (Editor)
This book offers an updated overview of the most recent research advances in the field, a comparison of established techniques and methods, a discussion on current experimental and translational challenges, and a commentary on potential opportunities and future directions.
Dedicated chapters address and review the preclinical and clinical state-of-the-art of gene therapies for the reconstructive and regenerative surgery of skin and wounds, pathological scars, cartilage, tendons, skeletal muscles, and bio-engineered flaps. A brief guide to developing gene therapy clinical trials in the... View Details
Advanced Textbook on Gene Transfer, Gene Therapy and Genetic Pharmacology: Principles, Delivery and Pharmacological and Biomedical Applications of ... (Icp Textbooks in Biomolecular Sciences)
by Daniel Scherman (Author), Daniel Scherman (Editor)
This unique advanced textbook provides a clear and comprehensive description of the field of gene delivery, gene therapy and genetic pharmacology, with descriptions of the main gene transfer vectors and a set of selected therapeutic applications, along with safety considerations.
The use of gene transfer is exponential in the scientific and medical community, both for cell biology experiments and for gene therapy revolutionary strategies. The advanced textbook should thus be of invaluable utility to Master, PhD or MD students, post-doctoral scientists or medical doctors, and to any... View Details
by Mary Colavito (Author), Michael A. Palladino (Author)
KEY MESSSAGE: Will gene therapy one day become the ultimate application of genetic technology? Gene Therapy, the newest booklet in the Special Topics in Biology Series, addresses this question by exploring gene therapy as a complex technology for delivering therapeutic genes as a way to treat and cure human genetic diseases. Author Mary Colavito provides an overview of the basic science involved in gene therapy methods and chronicles the history of gene therapy by discussing successful and ongoing gene therapy treatments as well as adverse outcomes in... View Details
Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition
by Nancy Smyth Templeton (Editor)
The Most Comprehensive, State-of-the-Art Book on Using Gene and Cell Therapy in Clinical Medicine
Gene and Cell Therapy: Therapeutic Mechanisms and Strategies, Fourth Edition presents extensive background and basic information, state-of-the-art technologies, important achievements, and lingering challenges in the fields of gene and cell therapies. The fourth edition of this bestseller continues to provide the most comprehensive coverage of these fields in one volume. Some chapters have expanded introductions, making the book even more suitable for classroom... View Details
Gene Therapy (Medical Marvels)
by L. E. Carmichael (Author)
Explores gene therapy, discussing how it works, how it was developed, applications of the technology, and where science will take the field in the future. View Details
A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution
by Jennifer A. Doudna (Author), Samuel H. Sternberg (Author)
A trailblazing biologist grapples with her role in the biggest scientific discovery of our era: a cheap, easy way of rewriting genetic code, with nearly limitless promise and peril.
Not since the atomic bomb has a technology so alarmed its inventors that they warned the world about its use. Not, that is, until the spring of 2015, when biologist Jennifer Doudna called for a worldwide moratorium on the use of the new gene-editing tool CRISPR—a revolutionary new technology that she helped create—to make heritable changes in human embryos. The cheapest,... View Details