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Tag: Gene Therapy
Gene scissors against incurable muscular disease
New gene correction therapy for Duchenne muscular dystrophy
Hundreds of UCLA students publish encyclopedia of 1,000 genes linked to organ development
A team of 245 UCLA undergraduates and 31 high school students has published an encyclopedia of more than 1,000 genes, including 421 genes whose functions were previously unknown. The research was conducted in fruit flies, and the genes the researchers…
Gene scissors against incurable muscular disease
New gene correction therapy for Duchenne muscular dystrophy
Hundreds of UCLA students publish encyclopedia of 1,000 genes linked to organ development
A team of 245 UCLA undergraduates and 31 high school students has published an encyclopedia of more than 1,000 genes, including 421 genes whose functions were previously unknown. The research was conducted in fruit flies, and the genes the researchers…
30-year study identifies need of disease-modifying therapies for maple syrup urine disease
A new study analyzes 30 years of patient data and details the clinical course of 184 individuals with genetically diverse forms of maple syrup urine disease
Researchers uncover mechanism for how common gene therapy vectors enter cells
Identification of GPR108 as ‘lock’ for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies
‘To safeguard people from chemical pollution, another approach is warranted’
We live in an increasingly complex world when it comes to chemicals. The number of new chemicals has increased from 20 million in 2002 to 156 million last year. Many of these are ubiquitous in the world around us because…
Researchers uncover mechanism for how common gene therapy vectors enter cells
Identification of GPR108 as ‘lock’ for adeno-associated virus vector (AAV) is crucial to this emerging novel class of genetic medicine and may provide framework for developing more targeted gene therapies
‘To safeguard people from chemical pollution, another approach is warranted’
We live in an increasingly complex world when it comes to chemicals. The number of new chemicals has increased from 20 million in 2002 to 156 million last year. Many of these are ubiquitous in the world around us because…
UMMS scientist shows gene therapy protection of eyesight in models of multiple sclerosis
Research identifies mechanism of synaptic damage in certain neurodegenerative disorders
Possible Parkinson’s treatment successfully targets two major nerve systems
Scientists have discovered that a non-invasive technique which could one day be used to treat Parkinson’s disease, can successfully target a highly specific group of brain cells which play a key role in development of the condition. In 2015, scientists…
Cardiac and visual degeneration arrested by a food supplement
UNIGE researchers have discovered a new gene that causes blindness and cardiomyopathy; they have also managed to halt the progression of eye disease and treat cardiac disease by administering a food supplement
UMMS scientist shows gene therapy protection of eyesight in models of multiple sclerosis
Research identifies mechanism of synaptic damage in certain neurodegenerative disorders
Possible Parkinson’s treatment successfully targets two major nerve systems
Scientists have discovered that a non-invasive technique which could one day be used to treat Parkinson’s disease, can successfully target a highly specific group of brain cells which play a key role in development of the condition. In 2015, scientists…
Cardiac and visual degeneration arrested by a food supplement
UNIGE researchers have discovered a new gene that causes blindness and cardiomyopathy; they have also managed to halt the progression of eye disease and treat cardiac disease by administering a food supplement
Partnership with China prompts change in care for high-risk type of leukemia
Findings from a collaborative clinical trial have generated key insights into how targeted therapy should be used to treat leukemia driven by the Philadelphia chromosome
Partnership with China prompts change in care for high-risk type of leukemia
Findings from a collaborative clinical trial have generated key insights into how targeted therapy should be used to treat leukemia driven by the Philadelphia chromosome
Partnership with China prompts change in care for high-risk type of leukemia
Findings from a collaborative clinical trial have generated key insights into how targeted therapy should be used to treat leukemia driven by the Philadelphia chromosome
Study shows lactate may prompt cancer formation
The byproduct of glucose may be catalyst that turns mutated cells to cancer
Study shows lactate may prompt cancer formation
The byproduct of glucose may be catalyst that turns mutated cells to cancer
Scientists breach brain barriers to attack tumors
The brain is a sort of fortress, equipped with barriers designed to keep out dangerous pathogens. But protection comes at a cost: These barriers interfere with the immune system when faced with dire threats such glioblastoma, a deadly brain tumor…
Scientists breach brain barriers to attack tumors
The brain is a sort of fortress, equipped with barriers designed to keep out dangerous pathogens. But protection comes at a cost: These barriers interfere with the immune system when faced with dire threats such glioblastoma, a deadly brain tumor…
Allon M. Klein receives Dr. Susan Lim Award for Outstanding Young Investigator
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award the Dr. Susan Lim Award for Outstanding Young Investigator to Allon M. Klein, PhD, Associate Professor, Systems Biology, Harvard Medical School, Boston, Mass., U.S. The prize recognizes the exceptional…
Fred H. Gage receives the ISSCR Achievement Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award its inaugural Achievement Award to Fred H. Gage, Ph.D., president of the Salk Institute for Biological Studies and the Vi and John Adler Chair for Research on Age-Related Neurodegenerative…
Margaret A. Goodell earns Tobias Lecture Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award the Tobias Lecture Award to Margaret A. (Peggy) Goodell, PhD, Chair of the Department of Molecular and Cellular Biology and Director of the Stem Cell and Regenerative Medicine Center…
Mitinori Saitou earns the ISSCR Momentum Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) present this year’s ISSCR Momentum Award to Mitinori Saitou, MD, Ph.D., Kyoto University, Japan. The prize recognizes the exceptional achievements of an investigator whose innovative research has established a major area…
Susan Solomon earns the ISSCR Public Service Award
Skokie, IL–Susan L. Solomon, JD, chief executive officer and founder of The New York Stem Cell Foundation (NYSCF) Research Institute is the recipient of the 2020 International Society for Stem Cell Research (ISSCR) Public Service Award. The honor recognizes outstanding…
Michael J. Bamshad, MD, named inaugural Editor-in-Chief of HGG Advances
New open access journal to launch in 2020
Allon M. Klein receives Dr. Susan Lim Award for Outstanding Young Investigator
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award the Dr. Susan Lim Award for Outstanding Young Investigator to Allon M. Klein, PhD, Associate Professor, Systems Biology, Harvard Medical School, Boston, Mass., U.S. The prize recognizes the exceptional…
Fred H. Gage receives the ISSCR Achievement Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award its inaugural Achievement Award to Fred H. Gage, Ph.D., president of the Salk Institute for Biological Studies and the Vi and John Adler Chair for Research on Age-Related Neurodegenerative…
Margaret A. Goodell earns Tobias Lecture Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) will award the Tobias Lecture Award to Margaret A. (Peggy) Goodell, PhD, Chair of the Department of Molecular and Cellular Biology and Director of the Stem Cell and Regenerative Medicine Center…
Mitinori Saitou earns the ISSCR Momentum Award
Skokie, IL–The International Society for Stem Cell Research (ISSCR) present this year’s ISSCR Momentum Award to Mitinori Saitou, MD, Ph.D., Kyoto University, Japan. The prize recognizes the exceptional achievements of an investigator whose innovative research has established a major area…
Susan Solomon earns the ISSCR Public Service Award
Skokie, IL–Susan L. Solomon, JD, chief executive officer and founder of The New York Stem Cell Foundation (NYSCF) Research Institute is the recipient of the 2020 International Society for Stem Cell Research (ISSCR) Public Service Award. The honor recognizes outstanding…
Michael J. Bamshad, MD, named inaugural Editor-in-Chief of HGG Advances
New open access journal to launch in 2020
Research identifies new route for tackling drug resistance in skin cancer cells
Researchers have found that melanoma cells fight anti-cancer drugs by changing their internal skeleton (cytoskeleton) – opening up a new therapeutic route for combatting skin and other cancers that develop resistance to treatment. The team, led by Queen Mary University…
Research identifies new route for tackling drug resistance in skin cancer cells
Researchers have found that melanoma cells fight anti-cancer drugs by changing their internal skeleton (cytoskeleton) – opening up a new therapeutic route for combatting skin and other cancers that develop resistance to treatment. The team, led by Queen Mary University…
Research identifies new route for tackling drug resistance in skin cancer cells
Researchers have found that melanoma cells fight anti-cancer drugs by changing their internal skeleton (cytoskeleton) – opening up a new therapeutic route for combatting skin and other cancers that develop resistance to treatment. The team, led by Queen Mary University…
Corning Life Sciences to showcase expanded portfolio of advanced 3D cell culture innovations
New solutions shared at SLAS 2020 support spheroid and organoid culture models and cell therapy research
Genethon announces expanded collaboration with Sarepta Therapeutics
Genethon announces expanded collaboration with Sarepta Therapeutics for continued development of an innovative gene therapy for Duchenne muscular dystrophy
Genethon announces expanded collaboration with Sarepta Therapeutics
Genethon announces expanded collaboration with Sarepta Therapeutics for continued development of an innovative gene therapy for Duchenne muscular dystrophy
UNH researchers solve protein structure associated with inherited retinal diseases
DURHAM, N.H. – Researchers at the University of New Hampshire have reported the first structural model for a key enzyme, and its activating protein, that can play a role in some genetically inherited eye diseases like retinitis pigmentosa and night…
UNH researchers solve protein structure associated with inherited retinal diseases
DURHAM, N.H. – Researchers at the University of New Hampshire have reported the first structural model for a key enzyme, and its activating protein, that can play a role in some genetically inherited eye diseases like retinitis pigmentosa and night…
Immune cell discovery opens door to new powerful cancer therapies
Scientists at UCL have identified how a subset of immune cells are activated to kill cancerous cells, a finding in mice which could hold the key to new powerful therapies against cancer. This new study built on previous research*, also…
Mayo Clinic researchers pursue single-dose gene therapy to treat cocaine addiction
New Rochelle, NY, January 7, 2020–In a radical new approach to treat cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose gene therapy. To support the safety and efficacy of this approach they…
Immune cell discovery opens door to new powerful cancer therapies
Scientists at UCL have identified how a subset of immune cells are activated to kill cancerous cells, a finding in mice which could hold the key to new powerful therapies against cancer. This new study built on previous research*, also…
Mayo Clinic researchers pursue single-dose gene therapy to treat cocaine addiction
New Rochelle, NY, January 7, 2020–In a radical new approach to treat cocaine addition, researchers at the Mayo Clinic are seeking approval for first-in-human studies of a single-dose gene therapy. To support the safety and efficacy of this approach they…
Gene therapy shown to offer long-term benefits for people with Haemophilia A
A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK. Clinical researchers led by Professor John Pasi from Queen Mary University of London…
Gene therapy shown to offer long-term benefits for people with Haemophilia A
A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK. Clinical researchers led by Professor John Pasi from Queen Mary University of London…
Injection of Virus-Delivered Gene Silencer Blocks ALS Degeneration, Saves Motor Function
Novel spinal therapy/delivery approach prevented disease onset in neurodegenerative ALS disease model in adult mice and blocked progression in animals already showing disease symptoms.