Category Archives: Gene therapy

When Google CEO Larry Page blogged about his struggles speaking and, at times, breathing last week on his Google+ page he spotlighted a rare condition, bilateral vocal cord paralysis , which leaves sufferers short of breath and with few viable treatment options. This is likely to change in coming years. Page has deep pockets and has promised to fund research into the disorder via the Voice Health Institute . In the meantime scientists are experimenting with electrical stimulation technologies to enhance existing voice therapy as well as surgical treatments. [More]












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http://rss.sciam.com/click.phdo?i=b3c657c36bd85f3126649923bb8701fe

Authors: Yoshida T, Kondoh M, Mizuguchi H, Yagi K
Abstract
Hepatitis C virus (HCV) is a hepatotropic member of the Flaviviridae family and contains a 9.6 kb positive-sense RNA genome. Approximately 170-million people are infected with HCV worldwide. These people face increased risks of chronic hepatitis, cirrhosis and hepatocellular carcinoma compared with the general population. Transduction of the HCV genome into hepatocytes is essential for understanding the mode of action of HCV infection, and for preparing HCV, evaluating HCV replication, and screening anti-HCV drugs. Although electroporation of in vitro-synthesized HCV genome and transduction of plasmid vectors containing the HCV genome are widely used in HCV research, a more convenient system with higher transduction efficie...

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http://www.medworm.com/index.php?rid=7281310&cid=c_449_13_f&fid=36240&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2Fpubmed%2F23449406%3Fdopt%3DAbstract

Authors: Yoshida T, Kondoh M, Mizuguchi H, Yagi K
Abstract
Hepatitis C virus (HCV) is a hepatotropic member of the Flaviviridae family and contains a 9.6 kb positive-sense RNA genome. Approximately 170-million people are infected with HCV worldwide. These people face increased risks of chronic hepatitis, cirrhosis and hepatocellular carcinoma compared with the general population. Transduction of the HCV genome into hepatocytes is essential for understanding the mode of action of HCV infection, and for preparing HCV, evaluating HCV replication, and screening anti-HCV drugs. Although electroporation of in vitro-synthesized HCV genome and transduction of plasmid vectors containing the HCV genome are widely used in HCV research, a more convenient system with higher transduction efficie...

Source:
http://www.medworm.com/index.php?rid=7281310&cid=c_449_13_f&fid=36240&url=http%3A%2F%2Fwww.ncbi.nlm.nih.gov%2Fpubmed%2F23449406%3Fdopt%3DAbstract

When Google CEO Larry Page blogged about his struggles speaking and, at times, breathing last week on his Google+ page he spotlighted a rare condition, bilateral vocal cord paralysis , which leaves sufferers short of breath and with few viable treatment options. This is likely to change in coming years. Page has deep pockets and has promised to fund research into the disorder via the Voice Health Institute . In the meantime scientists are experimenting with electrical stimulation technologies to enhance existing voice therapy as well as surgical treatments. [More]












Source:
http://rss.sciam.com/click.phdo?i=b3c657c36bd85f3126649923bb8701fe

Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis
A presentation by Stephanie Cherqui, PhD, University of California, San Diego at the 2013 Day of Hope Cystinosis Research Foundation Family Conference, Balbo...

By: Natalieswish

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Bone Marrow Stem Cell Transplantation and Gene Therapy for Cystinosis - Video

BOGOTA, Colombia, April 12, 2013 /PRNewswire/ -- Startup biotechnology company Neuralgene (http://neuralgene.com) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS.

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-a )

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-b )

Neuralgene's neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by Jason Williams, M.D., founder and CEO of Neuralgene. "This technology addresses several key aspects of the underlying pathology of ALS," said Leonardo Gonzalez, M.D., clinical researcher for Neuralgene. "In his stem cell work, Dr. Williams had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action."

The gene therapy is based on Dr. Williams' discovery that certain proteins produced by stem cells inhibit the attack of ALS. During the development of the gene therapy, he added new targets: neural growth factors and a protein implicated in ALS named TDP-43. "When Dr. Williams demonstrated the concept behind stem cells and how to address the treatment of ALS using gene therapy, we immediately knew that this was a revolutionary new concept," said Dr. Gonzalez.

The PRCN-829 gene therapy is designed to not only target gene delivery to the brain and spinal cord, but also to genetically engineer stem cells. The AAV9 viral vector delivers multiple genes, which include Factor H (a regulator of complement activity), neural growth factors and regulators of TDP-43, to the neural cells. Initial animal studies have demonstrated the safety of the gene therapy platform.

"The problem with stem cell therapy for ALS is that the results are generally partial and temporary," stated Dr. Williams. "This is because the stem cells produce the growth factors and other proteins for a short period, but then cease. Several stem cell studies have confirmed this. Now with gene therapy, we can increase those factors by a millionfold or greater so that recuperation lasts for many years or maybe is even lifelong."

"ALS is a complex disease with many different underlying causes," continued Dr. Williams. "Our gene therapy will target several of the main underlying mechanisms related to ALS with the hopes of getting a good response in a larger group of patients. However, our platform is versatile, allowing us to change and add different target genes. We expect that soon we will be able to perform a detailed genetic analysis of the patient, identifying their exact underlying cause of ALS. Then we will be able to tailor the therapy to each individual patient."

The company has partnered with several labs for the development of its patent-pending neurotropic AAV-based gene therapy for the treatment of neurodegenerative diseases such as ALS. In fact, Neuralgene partnered with Dr. Williams' imaging and image guided treatment facility, Precision StemCell in Bogota, to begin studies using image-guided administration of gene therapy to the spinal cord.

"This is a completely new therapy for ALS, and the groundwork for this technology will lead to the treatment of many other diseases," said Dr. Williams. Neuralgene has several other AAV gene therapies in its research and development pipeline for the treatment of neurodegenerative diseases such as Parkinson's and Multiple Sclerosis (MS). After initial testing of PRCN-829 in Colombia, Neuralgene plans to seek approval from the FDA for trials in the United States.

Link:
Gene Therapy Developed for ALS Treatment: New Biotech Company Neuralgene Enters Evaluation Phase

Mar. 25, 2013 In an animal study, researchers at the University of Washington show that it was possible to use gene therapy to boost heart muscle function. The finding suggests that it might be possible to use this approach to treat patients whose hearts have been weakened by heart attacks and other heart conditions.

Led by University of Washington (UW) Professor and Vice Chair of Bioengineering Michael Regnier and Dr. Chuck Murry, director of the Center for Cardiovascular Biology and co-director of the Institute for Stem Cell and Regenerative Medicine at UW, the study appears online today in the journal Proceedings of the National Academy of Sciences (PNAS).

Normally, muscle contraction is powered by a molecule, the nucleotide called Adenosine-5'-triphosphate (ATP). Other naturally occurring nucleotides can also power muscle contraction, but, in most cases, they have proven to be less effective than ATP.

In an earlier study of isolated muscle, however, Regnier, Murry and colleagues had found that one naturally occurring molecule, called 2 deoxy-ATP (dATP), was actually more effective than ATP in powering muscle contraction, increasing both the speed and force of the contraction, at least over the short-term.

In the new PNAS study, the researchers wanted to see whether this effect could be sustained. To do this, they used genetic engineering to create a strain of mice whose cells produced higher-than-normal levels of an enzyme called Ribonucleotide Reductase, which converts the precursor of ATP, adenosine-5'-diphosphate or ADP, to dADP, which, in turn, is rapidly converted to dATP.

"This fundamental discovery, that dATP can act as a 'super-fuel' for the contractile machinery of the heart, or myofilaments, opens up the possibility to treat a variety of heart failure conditions," Regnier said. "An exciting aspect of this study and our ongoing work is that a relatively small increase in dATP in the heart cells has a big effect on heart performance."

The researchers found that increased production of the enzyme Ribonucleotide Reductase increased the concentration of dATP within heart cells approximately tenfold, and even though this level was still less than one to two percent of the cell's total pool of ATP, the increase led to a sustained improvement in heart muscle function, with the genetically engineered hearts contracting more quickly and with greater force.

"It looks as though we may have stumbled on an important pathway that nature uses to regulate heart contractility," Murry added. "The same pathway that heart cells use to make the building blocks for DNA during embryonic growth makes dATP to supercharge contraction when the adult heart is mechanically stressed."

Importantly, the elevated dATP effect was achieved without imposing additional metabolic demands on the cells, suggesting the modification would not harm the cell's functioning over the long-term.

The finding, the authors write, suggest that treatments that elevate dATP levels in heart cells may prove to be an effective treatment for heart failure.

Continue reading here:
Gene therapy may aid failing hearts

Washington, Mar. 27 (ANI): Researchers have shown that it is possible to use gene therapy to treat patients whose hearts have been weakened by cardiac arrests and other heart conditions.

The research group was led by University of Washington (UW) Professor and Vice Chair of Bioengineering Michael Regnier and Dr. Chuck Murry, director of the Center for Cardiovascular Biology and co-director of the Institute for Stem Cell and Regenerative Medicine at UW.

Normally, muscle contraction is powered by a molecule, the nucleotide called Adenosine-5'-triphosphate (ATP).

In a previous study of isolated muscle, Regnier, Murry and colleagues had found that one naturally occurring molecule, called 2 deoxy-ATP (dATP), was actually more effective than ATP in boosting muscle contraction, increasing both the speed and force of the contraction, at least over the short-term.

In the new study, the researchers wanted to see if this effect could be sustained. For this, they used genetic engineering to create a strain of mice whose cells produced higher-than-normal levels of an enzyme called Ribonucleotide Reductase that converts the precursor of ATP, adenosine-5'-diphosphate or ADP, to dADP, which, in turn, is rapidly converted to dATP.

The researchers found that increased production of the enzyme Ribonucleotide Reductase increased the concentration of dATP within heart cells approximately tenfold, and even though this level was still less than one to two percent of the cell's total pool of ATP, the increase led to a sustained improvement in heart muscle function, with the genetically engineered hearts contracting more quickly and with greater force.

"The same pathway that heart cells use to make the building blocks for DNA during embryonic growth makes dATP to supercharge contraction when the adult heart is mechanically stressed," Murry said.

Importantly, the elevated dATP effect was achieved without imposing additional metabolic demands on the cells, suggesting the modification would not harm the cell's functioning over the long-term.

The findings suggest that treatments that elevate dATP levels in heart cells may prove to be an effective treatment for heart failure.

The study has been published in the journal Proceedings of the National Academy of Sciences (PNAS). (ANI)

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Gene therapy may help restore heart function after attack

Public release date: 21-Mar-2013 [ | E-mail | Share ]

Contact: Sarah Barth s.barth@elsevier.com 215-239-6087 Elsevier

Philadelphia, PA, March 21, 2013 The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured. Some of these disorders are exceedingly rare, others more common. The approach of gene therapy however may be applicable to all.

"The thirteen articles included in this special issue of Translational Research provide critical examples of the tools and practice of gene therapy today. They all focus on clinically meaningful studies that combine patient observations with smart experiments. The authors hope these articles will facilitate conversion of individual and disease-specific insight into a collective understanding of emerging gene transfer platforms and their subsequent translation to the bedside," explained contributing author Dr. Jakub Tolar of the Stem Cell Institute and Pediatric Blood and Marrow Transplant Program at the University of Minnesota, in his introduction to the issue. "The concept of gene therapy for genetic disorders is one of the most appealing in biomedicine because it is aimed at the cause rather than the symptoms of the disease."

Each article of this issue focuses on either a specific condition or a delivery method. Article topics included are: arthritis gene therapy, immunotherapies for type 1 diabetes mellitus, immune responses in liver-directed, lentiviral gene therapy, gene therapy for retinal disease, gene therapy in cystic fibrosis, evaluating risks of insertional mutagenesis by DNA transposons in gene therapy, pluripotent stem cells and gene therapy, gene therapy for hemoglobinopathies: progress and challenge, hemophilia clinical gene therapy-brief review, gene transfer for congestive heart failure, gene therapy for the prevention of vein graft disease, gene therapy for brain tumors, oncolytic virus therapy for cancer, and T cell-based gene therapy of cancer.

With the publication of this special issue, Translational Research identifies a need for clinical trial coordination among researchers worldwide, a focused goal of a world-scale change in medical practice, and real-time data exchange and evaluation, With these elements in place the true potential of gene therapy to treat and cure disease becomes apparent.

###

Notes for Editors

The articles appear in Translational Research, Volume 160, Issue 5 (April 2013), titled "Gene Therapy for Human Disease: Clinical Advances and Challenges," published by Elsevier, now available on ScienceDirect.

Full text of the articles included in the special issue is available to credentialed journalists upon request. Contact Sarah Barth at +1 215 239 6087, s.barth@elsevier.com to obtain copies or to schedule an interview with Dr. Jeffrey Laurence, MD, Editor-in-Chief.

See the rest here:
' Gene Therapy for Human Disease: Clinical Advances and Challenges'

Public release date: 21-Mar-2013 [ | E-mail | Share ]

Contact: Sarah Barth s.barth@elsevier.com 215-239-6087 Elsevier

Philadelphia, PA, March 21, 2013 The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured. Some of these disorders are exceedingly rare, others more common. The approach of gene therapy however may be applicable to all.

"The thirteen articles included in this special issue of Translational Research provide critical examples of the tools and practice of gene therapy today. They all focus on clinically meaningful studies that combine patient observations with smart experiments. The authors hope these articles will facilitate conversion of individual and disease-specific insight into a collective understanding of emerging gene transfer platforms and their subsequent translation to the bedside," explained contributing author Dr. Jakub Tolar of the Stem Cell Institute and Pediatric Blood and Marrow Transplant Program at the University of Minnesota, in his introduction to the issue. "The concept of gene therapy for genetic disorders is one of the most appealing in biomedicine because it is aimed at the cause rather than the symptoms of the disease."

Each article of this issue focuses on either a specific condition or a delivery method. Article topics included are: arthritis gene therapy, immunotherapies for type 1 diabetes mellitus, immune responses in liver-directed, lentiviral gene therapy, gene therapy for retinal disease, gene therapy in cystic fibrosis, evaluating risks of insertional mutagenesis by DNA transposons in gene therapy, pluripotent stem cells and gene therapy, gene therapy for hemoglobinopathies: progress and challenge, hemophilia clinical gene therapy-brief review, gene transfer for congestive heart failure, gene therapy for the prevention of vein graft disease, gene therapy for brain tumors, oncolytic virus therapy for cancer, and T cell-based gene therapy of cancer.

With the publication of this special issue, Translational Research identifies a need for clinical trial coordination among researchers worldwide, a focused goal of a world-scale change in medical practice, and real-time data exchange and evaluation, With these elements in place the true potential of gene therapy to treat and cure disease becomes apparent.

###

Notes for Editors

The articles appear in Translational Research, Volume 160, Issue 5 (April 2013), titled "Gene Therapy for Human Disease: Clinical Advances and Challenges," published by Elsevier, now available on ScienceDirect.

Full text of the articles included in the special issue is available to credentialed journalists upon request. Contact Sarah Barth at +1 215 239 6087, s.barth@elsevier.com to obtain copies or to schedule an interview with Dr. Jeffrey Laurence, MD, Editor-in-Chief.

Read more:
' Gene Therapy for Human Disease: Clinical Advances and Challenges'