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Archive for the ‘Gene Therapy Research’ Category

Report of the American Society of Human Genetics Ancestry Inference Roundtable (Panel) – Video


Report of the American Society of Human Genetics Ancestry Inference Roundtable (Panel)
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

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Report of the American Society of Human Genetics Ancestry Inference Roundtable (Panel) - Video

Identity and Genetics (Introduction) – Yolanda Moses – Video


Identity and Genetics (Introduction) - Yolanda Moses
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

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Identity and Genetics (Introduction) - Yolanda Moses - Video

DNA GENETICS AMSTERDAM 2013 – Video


DNA GENETICS AMSTERDAM 2013
DNA GENETICS AMSTERDAM http://www.dnagenetics.com DNA Genetics Sint Nicolaasstraat 41 Amsterdam TheHashCompany Cannabis Entertainment Amsterdam http://www....

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DNA GENETICS AMSTERDAM 2013 - Video

A Nurse’s Testament on Adult Stem Cell Therapy for Back Pain – Video


A Nurse #39;s Testament on Adult Stem Cell Therapy for Back Pain
A registered nurse describes her experience with an adult stem cell therapy procedure for back pain. More information at medrebels.org.

By: Med Rebels

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A Nurse's Testament on Adult Stem Cell Therapy for Back Pain - Video

News Release: Dr. Andrew Cappuccino’s Insight on Adult Stem Cell Therapy – Video


News Release: Dr. Andrew Cappuccino #39;s Insight on Adult Stem Cell Therapy
Dr. Andew Cappuccino, team orthopedist for the Buffalo Bills, gives insight on using Adult Stem Cells to treat back pain. More information at http://medrebel...

By: Med Rebels

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News Release: Dr. Andrew Cappuccino's Insight on Adult Stem Cell Therapy - Video

Public Lecture; Kevin Mitchell, Smurfit Institute of Genetics, Trinity College Dublin – Video


Public Lecture; Kevin Mitchell, Smurfit Institute of Genetics, Trinity College Dublin
"The Miswired Brain" Public Lecture 7/17/2013.

By: CSHL Leading Strand

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Public Lecture; Kevin Mitchell, Smurfit Institute of Genetics, Trinity College Dublin - Video

Great young lyretail genetics, – Video


Great young lyretail genetics,
2 that are lyretails that show 1 red with out lyretail showing but of same genetics as the 2 above remaing are of the same genetics but black.

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Great young lyretail genetics, - Video

Mom’s gene therapy interview – Video


Mom #39;s gene therapy interview
Jake #39;s gene therapy interview with mom.

By: Julie Perine

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Mom's gene therapy interview - Video

Alliance for Cancer Gene Therapy- Full Program at Harvard Club on October 15,2013 – Video


Alliance for Cancer Gene Therapy- Full Program at Harvard Club on October 15,2013
Alliance for Cancer Gene Therapy #39;s top researchers and physicians discuss the success of gene and cell therapy at the Harvard Club on October 15, 2013.

By: Matthew Lagle

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Alliance for Cancer Gene Therapy- Full Program at Harvard Club on October 15,2013 - Video

Report of the American Society of Human Genetics Ancestry (Introduction) – Vence Bonham – Video


Report of the American Society of Human Genetics Ancestry (Introduction) - Vence Bonham
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

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Report of the American Society of Human Genetics Ancestry (Introduction) - Vence Bonham - Video

Population Genetics in the Personal Genome Era – Carlos Bustamante – Video


Population Genetics in the Personal Genome Era - Carlos Bustamante
September 12, 2013 - The African Diaspora: Integrating Culture, Genomics and History.

By: GenomeTV

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Population Genetics in the Personal Genome Era - Carlos Bustamante - Video

Gene therapy of adrenoleukodistrophy – Video


Gene therapy of adrenoleukodistrophy
Dra. Nathalie Cartier.

By: AsociacionAMER

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Gene therapy of adrenoleukodistrophy - Video

the Gene Therapy Center – University of North Carolina at Chapel …

The University of North Carolina School of Medicine created the Gene Therapy Center in 1996 with the goal of merging molecular genetics research with healthcare delivery. The Gene Therapy Center provides important resources to academic investigators through two core facilities created to support preclinical and clinical gene therapy studies. These facilities, the Vector Core and the Human Applications Laboratories, were created to ensure that investigators would have promising gene vectors available in the quality and quantities needed for preclinical or clinical studies. Research in the laboratory has centered on the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy. Continued efforts in understanding the mechanism of viral replication and integration for both wild-type and recombinant AAV are being pursued in order to create more efficient gene transfer vectors. The ultimate goal of the Gene Therapy Center is to facilitate the progression and translation of gene therapy research from the laboratory bench into Phase I clinical trials for the treatment of human disease. Click here to learn more about our gene therapy research.

Announcements

February 2013

Symposium: The Nexus of Gene Therapy & Regenerative Medicine

Location: The Old Salem Visitor Center, Winston-Salem, NC

Date: Thursday, February 7, 2013 9AM - 6PM

October 2011

Gene Therapists Celebrate a Decade of Progress

http://www.sciencemag.org/content/334/6052/29.full

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the Gene Therapy Center - University of North Carolina at Chapel ...

stem cell therapy treatment for Quadriplegic Cerebral Palsy by dr alok sharma, mumbai, india – Video


stem cell therapy treatment for Quadriplegic Cerebral Palsy by dr alok sharma, mumbai, india
improvement seen in just 3 months after stem cell therapy treatment for quadriplegic cerebral palsy by dr alok sharma, mumbai, india. Stem Cell Therapy done ...

By: Neurogen Brain and Spine Institute

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stem cell therapy treatment for Quadriplegic Cerebral Palsy by dr alok sharma, mumbai, india - Video

Genentech – Official Site

New Genentech Medicine for Previously Untreated Chronic Lymphocytic Leukemia

At Genentech, our t-shirts represent our stories - about our people, our community, and ultimately about the impact of biotechnology on medicine.

Genentech is committed to ensuring price is not a barrier to access for patients. Learn about our programs.

Physicians can now use our secure online portal to review Sunshine-reportable payments before they are reported to the government.

Search for jobs. Learn more about what is currently available. And then apply for the position at Genentech thats right for you.

Genentech Disappointed with CA Governor's Veto of Patient Access to Biosimilars Bill

Insights on trends, events, and therapeutic advances. Learn more at Genentech-Forum.com.

Ann Lee-Karlon, Ph.D., SVP of Portfolio Management and operations on how teams and decision making influence drug discovery and development.

Our pipeline includes over 30 new molecular entities that address serious unmet medical needs.

Partnering is a relatively new phenomenon for biopharmaceutical companies, but collaboration has been part of Genentechs DNA from the beginning.

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Genentech - Official Site

Paringa Livestock Sheep Genetics Overview 2013 – Video


Paringa Livestock Sheep Genetics Overview 2013

By: clearvisionimaging

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Paringa Livestock Sheep Genetics Overview 2013 - Video

stem cell therapy treatment for dystonic cerebral palsy by dr alok sharma, mumbai, india – Video


stem cell therapy treatment for dystonic cerebral palsy by dr alok sharma, mumbai, india
improvement seen in just 3 months after stem cell therapy treatment for dystonic cerebral palsy by dr alok sharma, mumbai, india. Stem Cell Therapy done date...

By: Neurogen Brain and Spine Institute

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stem cell therapy treatment for dystonic cerebral palsy by dr alok sharma, mumbai, india - Video

O.M.D. – Genetic Engineering – Video


O.M.D. - Genetic Engineering

By: Countdown Chart History

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O.M.D. - Genetic Engineering - Video

Bridge To The Soul – Video


Bridge To The Soul
Art 4 All People in collaboration with USC present: Bridge To The Soul/The Art Of Healing art exhibition curated by the transformative arts organization, Art...

By: Beatriz Eugenia

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Bridge To The Soul - Video

Stem Cell Stories trailer – Stem Cell Therapy Europe – Video


Stem Cell Stories trailer - Stem Cell Therapy Europe
Stem Cell Stories trailer - Stem Cell Therapy Europe.

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Stem Cell Stories trailer - Stem Cell Therapy Europe - Video

Progenitor Cell Therapy | A NeoStem Company | Allendale, Mountain View

(201) 677-CELL

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At PCT, we're all about you, our Client. We're committed to cell therapy, our Clients, and their success. PCT is an industry leader in contract development and manufacturing of cell therapy products. With over 12 years of exclusive cell therapy-focused experience, we help our Clients bridge the gap between discovery and patient care through efficient transfer of cell-based therapies from laboratory into clinical practice. In all Client partnerships we place paramount importance on product quality, technical and operational excellence, exceptional customer service, and the protection of Client's proprietary information. We're here to make sure your dream manifests in the real world.LEARN MORE

PCT is dedicated to bridging the gap between the concepts in the cell therapy industry and their therapeutic application in patient care by providing our Clients with the maximum quality and value services in support of their cell therapy product and technology commercialization efforts.

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Progenitor Cell Therapy | A NeoStem Company | Allendale, Mountain View

Spinal Muscular Atrophy Gene Therapy Research

Overview of Gene Therapy for SMA

Updated September 20, 2013

Click here for a Drug Discovery Compass on Gene Therapy.

Click here for a summary article from Genzyme on the status of SMA Gene Therapy.

What is gene therapy? Gene Therapy is an approach to treating diseases by either modifying the expression of particular genes or by correcting abnormal genes. Gene therapy works by administration of DNA rather than a drug. Many different diseases are currently being investigated as candidates for gene therapy. These include cystic fibrosis, cardiovascular disease, AIDS, cancer and SMA.1

A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they cant cause disease when used in people. Viruses, such as adenoviruses, introduce their DNA into the cell. The gene therapy vector can be given systemically by IV or injected directly into a specific tissue in the body, where it is taken up by individual cells, like motor neurons in the case of SMA.

What has FSMA's role been in the project?-In 2010, FSMA awarded $100,000 to Dr. Brian Kaspar at Nationwide Childrens Hospital for SMA gene therapy development. Please click here to read the announcement. -In 2011, Dr. Brian Kaspar published a paper in "Molecular Therapy" on systemic gene delivery in large species with FSMA funding. Please click here to read the annoucement. -In 2012, FSMA awarded Dr. Kaspar $750,00 in drug discovery funding to advance a CNS delivered gene therapy for SMA. Please click here to read the announcement. -In 2013, NINDS awarded a $3,752,462 to Dr. Kaspar in collaboration with FSMA to advance a CNS-directed gene therapy to IND. Please click here to read the announcement.

How does gene therapy work in SMA? In the case of SMA, gene therapy could take several approaches. The most likely approach would be to replace the lost SMN1 gene in cells. A second approach could be to use small pieces of genetic material, called oligonucleotides, to improve the functioning of the back-up SMN2 gene. Please click here to read more about that approach. A third approach could be to use gene therapy to deliver neuroprotective proteins like growth factors to motor neurons to help keep them alive and well functioning.

The challenge with gene therapy for SMA is to find a way to deliver the genetic material to the spinal cord. A number of viruses are being studied for their ability to safely and effectively carrier the genetic material across the blood brain barrier, where they will be needed to treat a disease like SMA. In the past, these viruses could not cross the blood brain barrier. Recently one called AAV9 has been generated that reaches motor neurons very effectively. See figure below for details.

Figure 1. Gene therapy for mice with spinal muscular atrophy (SMA). SMA mice (null for the murine SMN gene and homozygous for variants of human SMN transgenes) are born with a normal motor neuron complement. However, the motor neurons undergo rapid attrition, likely a result of synaptic failure and denervation with attendant muscular atrophy. The mice become wasted and succumb at two weeks of age (left), analogous to an untreated mild human type I SMA. Injection of scAAV9-SMN into the facial vein of day-old SMA pups results in SMN expression in ~40% of motor neurons, normalization of synaptic electrophysiology and an extension of life span to >250 days, albeit at half the size of unaffected mice (right). Full results published in Foust, K.D. et al. Nat. Biotechnol. 28, 271274 (2010). This result has been replicated by multiple labs worldwide, including at Genzyme Corporation, the lab of Dr. Martine Barkats, and the lab of Dr. Mimoun Azzouz. Figure reprinted by permission from Macmillan Publishers Ltd: Nature Biotechnology, Mackenzie A., Genetic therapy for spinal muscular atrophy. Volume 28: 236-7. Copyright 2010.

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Spinal Muscular Atrophy Gene Therapy Research

Medical Educational apps QVprep Lite Learn Genetics and Genetic Engineering app video part 10 11 1 – Video


Medical Educational apps QVprep Lite Learn Genetics and Genetic Engineering app video part 10 11 1
QVprep Lite Genetic Engineering is FREE and has limited content. The app gives you the option to buy the paid QVprep Genetic Engineering app which has exhaus...

By: Deep Larry

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Medical Educational apps QVprep Lite Learn Genetics and Genetic Engineering app video part 10 11 1 - Video

Medical Educational Apps QVprep Lite Learn Genetic and genetic engineering app video part 6 7 8 9 – Video


Medical Educational Apps QVprep Lite Learn Genetic and genetic engineering app video part 6 7 8 9
QVprep Lite Genetic Engineering is FREE and has limited content. The app gives you the option to buy the paid QVprep Genetic Engineering app which has exhaus...

By: Deep Larry

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Medical Educational Apps QVprep Lite Learn Genetic and genetic engineering app video part 6 7 8 9 - Video

Medical Educational apps QVprep Learn genetics and Genetic Engineering app video part 13 14 – Video


Medical Educational apps QVprep Learn genetics and Genetic Engineering app video part 13 14
QVprep Lite Genetic Engineering is FREE and has limited content. The app gives you the option to buy the paid QVprep Genetic Engineering app which has exhaus...

By: Deep Larry

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Medical Educational apps QVprep Learn genetics and Genetic Engineering app video part 13 14 - Video

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