His laboratory (The Laboratory for
Cell and Gene Medicine) specializes in the development of manufacturing processes and QC assays and provides cGMP compliant clinical materials production and regulatory support activities for investigational cell products.
Baldrick's Foundation Pediatric Cancer Dream Team and Associate Director, Stanford Cancer Institute; Co-medical Director, Standford Laboratory for
Cell and Gene Medicine Dr. Gabriel Otte, Co-founder and CEO, Freenome Inc..
Improving human health by enabling safer, more effective
cell and gene medicines through proprietary genomics, bioinformatics and intelligent data driven design
Not exact matches
New technologies such as
gene and cell therapies hold out the potential to transform
medicine and create an inflection point in our ability to treat
and even cure many intractable illnesses.
Gene Yeo, a professor of cellular
and molecular
medicine at UCSD, led the research
and showed he could target RNA in living
cells, a first step toward treating diseases like muscular dystrophy
and neurodegeneration.
«It was kind of fun being at a medical school
and known as the weird guy who worked with dogs,» says Modiano, who is now a professor of comparative oncology at the University of Minnesota College of Veterinary
Medicine and the Masonic Cancer Center, where his research focuses on immunology, cancer
cell biology, cancer genetics,
and applications of
gene therapy.
«The
gene has been cloned,
and we know it interferes with the production of toxic amyloid fragments,» says Ralph Nixon, a professor of psychiatry
and cell biology at New York University School of
Medicine and a past chair of the Medical
and Scientific Advisory Council of the Alzheimer's Association.
Priscilla N. Kelly Associate Editor Education: B.Sc., University of Western Australia; Ph.D., University of Melbourne Areas of responsibility: Preclinical development, translational
medicine, cancer immunotherapy, drug discovery, clinical trials,
gene and cell therapy E-Mail:
[email protected]
In a 2006 study published in
Cell, Vidal
and Huda Zoghbi, a neurobiologist at Baylor College of
Medicine in Houston, Texas, teamed up to show previously unsuspected interactions among the proteins produced by mutated
genes in several of the movement disorders known as ataxias.
Researchers at the Center for
Cell and Gene Therapy at Baylor College of
Medicine, Texas Children's Hospital
and Houston Methodist have developed an alternative treatment in which virus - specific
cells protect patients against severe, drug - resistant viral infections.
«In our experiments, our nanoparticles successfully delivered a test
gene to brain cancer
cells in mice, where it was then turned on,» says Jordan Green, Ph.D., an assistant professor of biomedical engineering
and neurosurgery at the Johns Hopkins University School of
Medicine.
At the Center for
Cell and Gene Therapy at Baylor College of
Medicine in Houston, director Malcolm Brenner, a geneticist, says he has turned «AdV from poacher into gamekeeper.»
To better understand how HMGA1 affected the rodents» intestines, Resar
and Lingling Xian, M.D., Ph.D., research associate at the Johns Hopkins University School of
Medicine,
and their colleagues examined the transgenic animals» intestinal
cells to determine which ones were expressing this
gene.
In a report that appears in PLOS BIOLOGY, Dr. Hugo Bellen
and his colleagues at Baylor College of
Medicine and the Jan
and Dan Duncan Neurological Research Institute at Texas Children's Hospital
and BCM,
and Dr. Chao Tong, at the Life Sciences Institute
and Innovation Center for
Cell Biology, Zhejiang University in Hangzhou, China, find that mutations of human homologs (
genes that carry out similar functions) of cacophony
and its partner straightjacket (Cacna1a
and Cacna2d2 respectively) cause defects in autophagy in neurons.
One miRNA can target multiple
genes, but their expression is often hijacked by cancer
cells and disrupts multiple cancer - causing or tumor - suppressing pathways,» says Shuk - Mei Ho, PhD, director of the CCC
and Jacob G. Schmidlapp Chair of Environmental Health
and professor at the University of Cincinnati (UC) College of
Medicine.
Of particular interest are the emerging techniques for genomics
and proteomics, which allow profiles of
gene expression
and protein synthesis to be produced
and comparisons to be made between normal
and abnormal
cells, as well as between
cells before
and after exposure to
medicines or toxic chemicals.
In an effort to expand the number of cancer
gene mutations that can be specifically targeted with personalized therapies, researchers at University of California San Diego School of
Medicine and Moores Cancer Center looked for combinations of mutated
genes and drugs that together kill cancer
cells.
«Researchers ID cancer
gene - drug combinations ripe for precision
medicine: Yeast, human
cells and bioinformatics help develop one - two punch approach to personalized cancer therapy.»
«If you look at a set of lung cancer patients, like we did in the paper, who develop brain metastases, they all have those two
genes in their primary lung cancer,» said Sheila Singh, the study's supervisor, associate professor at the Michael G. DeGroote School of
Medicine, scientist with the Stem
Cell and Cancer Research Institute at McMaster University
and neurosurgeon at McMaster Children's Hospital.
In a study led by Eli
and Edythe Broad Center of Regenerative
Medicine and Stem
Cell Research member Dr. Julian Martinez - Agosto, UCLA scientists have shown that two
genes not previously known to be involved with the immune system play a crucial role in how progenitor stem
cells are activated to fight infection.
Myc is a cancer - causing
gene responsible for disrupting the normal 24 - hour internal rhythm
and metabolic pathways in cancer
cells, found a team led by researchers from the Perelman School of
Medicine at the University of Pennsylvania.
The team, which included members of the Health Science Center departments of
medicine and biochemistry, investigators from the UT Southwestern Medical Center at Dallas
and a group of collaborators from Austria, found that the
gene that codes the enzyme D2 - hydroxyglutarate dehydrogenase (D2HGDH) is mutated in a subset of cancers called diffuse large B -
cell lymphomas.
The studies on autophagy by Yoshinori Ohsumi, which earned him the Nobel Prize in
Medicine in 2016,
and the discovery of
cell cycle regulatory
genes for which Leland Hartwell, Timothy Hunt
and Paul Nurse received the same award in 2001, including the research of Elizabeth Blackburn, Carol Greider
and Jack Szostak on telomeres, telomerase
and its protective effect on the chromosomes, were all made possible thanks to yeast.
Rivals such as Pfizer
and Sanofi are also investing,
and overall financing for
gene and gene - modified
cell therapies reached $ 1 billion in the first quarter of 2017, according to the Alliance of Regenerative
Medicine.
«We found that MYSM1 creates access to proteins that enhance
gene transcription
and, ultimately, the maturation of natural killer
cells themselves,» said Vijayalakshmi Nandakumar, a Ph.D. student at the Keck School of
Medicine of USC
and the study's first author.
Researchers from the University of Seville at the Andalusian Centre for Molecular Biology
and Regenerative
Medicine (Centro Andaluz de Biología Molecular y Medicina Regenerativa — Cabimer) have discovered that in eukaryotic
cells the proximity of the
genes to the nuclear pores, which are found in the nuclear membrane, contributes to maintaining the integrity of the genome.
In a series of studies this year, molecular geneticists at the University of Pittsburgh School of
Medicine used a harmless virus to ferry new
genes through the bloodstream, across blood vessel walls,
and into almost every muscle
cell in the bodies of hamsters bred to have human genetic diseases.
For the animal experiments, Savio Woo of the Center for
Gene Therapy at Baylor College of
Medicine in Houston
and his colleagues first isolated liver
cells from transgenic mice that produce the human protein a1 - antitrypsin in their livers, from where it is secreted into the blood.
But when physiologist H. Lee Sweeney of the University of Pennsylvania School of
Medicine in Philadelphia
and his colleagues put this faulty
gene into embryonic quail muscle
cells growing in lab dishes, the
cells made a shortened version of the protein
and incorporated it into their contractile machinery.
Liangxue Lai of the South China Institute for Stem
Cell Biology
and Regenerative
Medicine in Guangzhou, China,
and colleagues injected the
gene editor into 35 beagle embryos.
«Molecular
Medicine BioServices makes viruses for companies working on vaccines,
gene,
and cell therapies.
Polyploid
cells, which carry additional copies of important tumor suppressor
genes, are better protected
and more resistant to cancer formation because they have these extra copies of the genome,» said Dr. Zhu, who is also an Assistant Professor of Pediatrics
and Internal
Medicine at UT Southwestern.
PHYSIOLOGY OR
MEDICINE: Andrew Z. Fire of the Stanford University School of
Medicine and Craig C. Mello of the University of Massachusetts Medical School, for their 1998 discovery of RNA interference, which regulates
gene expression
and helps defend
cells against viruses.
«In a nutshell, what we found is that this
gene has an effect on the frequency of these T follicular helper
cells, which are important for the B
cells and seem to be important for the disease,» says Dr. Kissler, who is also an assistant professor of
medicine at Harvard Medical School.
To probe the link between
gene and virus, neuroscientist HowardFederoff, of the University of Rochester School of
Medicine,
and his colleagues created mouse -
cell cultures that expressed either theAPOE - e4
gene or another variant (APOE - e2 or APOE - e3), then infected the
cells with herpes.
In today's issue of Science Translational
Medicine, he
and his colleagues present a more efficient way of finding such new uses for old drugs: by bringing together data on how diseases
and drugs affect the activity of the roughly 30,000
genes in a human
cell.
A physician with a longstanding interest in
gene therapy, Dr. High was formerly a Professor at the Perelman School of
Medicine of the University of Pennsylvania, an Investigator of the Howard Hughes Medical Institute,
and the Founding Director of the Center for Cellular
and Molecular Therapeutics at The Children's Hospital of Philadelphia, a Center focused on developing novel
cell and gene - based therapies for genetic disease.
Using a novel technique to genetically modify T
cells for adoptive transfer, Carl June, Michael Kalos, David Porter, Bruce Levine,
and colleagues at the University of Pennsylvania School of
Medicine achieve clinical responses in patients with chronic lymphocytic leukemia, including two complete, durable (one year) clinical responses, accompanied by in vivo expansion
and long - term functional persistence of
gene - modified
cells.
Now in experiments in mice reported this week in
Cell Metabolism, researchers at Joslin Diabetes Centers have highlighted the ways in which the host's
genes interact with the microbial
genes to create such conditions, says senior author C. Ronald Kahn, M.D., Chief Academic Officer at Joslin Diabetes Center
and Mary K. Iacocca Professor of
Medicine at Harvard Medical School.
Therapeutic applications of CRISPR - Cas
gene editing are being advanced with the goals of finding cures
and treatments for a range of serious diseases, developing needed
medicines for both household pets
and production animals,
and improving
cell culture systems for bioproduction.
Data from this trial was previously presented at World Advanced Therapies
and Regenerative
Medicine (May 17 - 19,2017; London, UK)
and the 20th Annual Meeting American Society of
Gene and Cell Therapy (ASGCT, May 10 - 13, 2017; DC, USA).
The 2017 Annual Data Report details industry - specific statistics
and trends from more than 850 leading
cell therapy,
gene therapy, tissue engineering,
and other regenerative
medicine companies worldwide.
He is Member of the European Molecular Biology Organization (EMBO), has been President of the European Society of
Gene and Cell Therapy (ESGCT),
and has been appointed as expert on the «Human
Gene Editing Study» of the US National Academies of Sciences
and of
Medicine,
and on the Italian National Committee for Biosafety, Biotechnology
and Life Sciences.
Supported by: Hear from leading expert Dr David DiGiusto, Stanford University School of
Medicine, USA, as he shares his extensive experience in developing
and optimizing manufacturing processes for
cell and gene therapy products including:
In the second study, a team led by Shahin Rafii at Weill Cornell
Medicine in New York City used adult mouse
cells as their starting material,
and then guided them through several steps — including exposure to some of the same
gene - activating proteins — to create mature blood stem
cells in a petri dish.
In a previous study, Shaw, an associate professor in Salk's Molecular
and Cell Biology Laboratory
and researcher in the Institute's new Helmsley Center for Genomic
Medicine, demonstrated that
cells lacking a normal copy of the LKB1
gene fail to activate AMPK in response to low energy levels.
«To everyone's jaw - dropping surprise, you needed only four
genes,» says stem
cell biologist David Scadden, director of the Center for Regenerative Medicine at the MGH and co-director of the Harvard Stem Cell Instit
cell biologist David Scadden, director of the Center for Regenerative
Medicine at the MGH
and co-director of the Harvard Stem
Cell Instit
Cell Institute.
Don't miss the chance to make new contacts, connect with current clients, develop your
cell therapy, regenerative
medicine and gene therapy business.
Chris is on a number of national
and international committees, working groups
and initiatives related to the academic, clinical translation
and commercialization of
cell and gene therapies including; Founder and CEO of the London Regenerative Medicine Network (LRMN), Founding Member of the UK - Israel Science Council, Scientific Advisory Board of the UK Cell and Gene Therapy Catapult, Strategic Advisory Board of the Canadian Centre for the Commercialization of Regenerative Medicine (CCRM), and Scientific Advisory Board of the Canadian Stem Cell Netw
cell and gene therapies including; Founder and CEO of the London Regenerative Medicine Network (LRMN), Founding Member of the UK - Israel Science Council, Scientific Advisory Board of the UK Cell and Gene Therapy Catapult, Strategic Advisory Board of the Canadian Centre for the Commercialization of Regenerative Medicine (CCRM), and Scientific Advisory Board of the Canadian Stem Cell Netw
gene therapies including; Founder
and CEO of the London Regenerative
Medicine Network (LRMN), Founding Member of the UK - Israel Science Council, Scientific Advisory Board of the UK
Cell and Gene Therapy Catapult, Strategic Advisory Board of the Canadian Centre for the Commercialization of Regenerative Medicine (CCRM), and Scientific Advisory Board of the Canadian Stem Cell Netw
Cell and Gene Therapy Catapult, Strategic Advisory Board of the Canadian Centre for the Commercialization of Regenerative Medicine (CCRM), and Scientific Advisory Board of the Canadian Stem Cell Netw
Gene Therapy Catapult, Strategic Advisory Board of the Canadian Centre for the Commercialization of Regenerative
Medicine (CCRM),
and Scientific Advisory Board of the Canadian Stem
Cell Netw
Cell Network.
Prior to that, Dr. Matushansky was at Novartis where he was Global Head for Clinical
and Scientific Development at its
Gene &
Cell Therapy Unit as well as a Global Clinical Program Lead within Novartis» Oncology Translational
Medicine Unit.