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UrduInvestigational drug reverses motor deficits in pre-clinical models, even administered one month after birth
Tag: Gene Therapy
In NIH trial, selumetinib shrinks tumors, provides clinical benefit for children with NF1
Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1 called plexiform neurofibromas, and…
St. Jude finds cancer drug resistance genes and possibly how to limit their effects
Drug resistance is a leading cause of cancer death in children and adults with leukemia. St. Jude Children’s Research Hospital scientists have developed a novel strategy to find the genes responsible
International study completes the largest genetic map of psychiatric disorders so far
A new genetic perspective on psychiatric disorders
Nature Gene Therapy publishes preclinical data of Ocugen’s OCU400 genetic modifier
Data support nuclear hormone receptor gene NR2E3 as genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases and potentially serve as a broad-spectrum therapy
Gene therapy generates new neurons to treat Huntington’s disease
Gene therapy to treat Huntington’s disease
Scientists successfully test new way to deliver gene therapy
Researchers at Case Western Reserve University, funded in part through a Gund-Harrington Scholar grant at University Hospitals in Cleveland, have used used chemically modified lipids—instead of the viruses most commonly used as carriers— to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease.
Cells carrying Parkinson’s mutation could lead to new model for studying disease
MADISON — Parkinson’s disease researchers have used gene-editing tools to introduce the disorder’s most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular chemistry that often goes awry in Parkinson’s patients. The edited cells are…
Cells carrying Parkinson’s mutation could lead to new model for studying disease
MADISON — Parkinson’s disease researchers have used gene-editing tools to introduce the disorder’s most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular chemistry that often goes awry in Parkinson’s patients. The edited cells are…
Roberts Individualized Medical Genetics Center outlines centralized genetic testing model
A pioneer in the field, CHOP-based Center brings together researchers, physicians, and genetic counselors to help patients navigate the complexities of genetic testing
Roberts Individualized Medical Genetics Center outlines centralized genetic testing model
A pioneer in the field, CHOP-based Center brings together researchers, physicians, and genetic counselors to help patients navigate the complexities of genetic testing
New CRISPR base-editing technology slows ALS progression in mice
CHAMPAIGN, Ill. — With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis – a debilitating and fatal neurological disease for which…
New CRISPR base-editing technology slows ALS progression in mice
CHAMPAIGN, Ill. — With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis – a debilitating and fatal neurological disease for which…
Plant-based relatives of cholesterol could give boost to gene therapy
PORTLAND, Ore. – Gene-infused nanoparticles used for combating disease work better when they include plant-based relatives of cholesterol because their shape and structure help the genes get where they need to be inside cells. The findings by Oregon State University…
Curing genetic disease in human cells
Utrecht, The Netherlands, 20th of February 2020. While the genome editing tool CRISPR/Cas9, developed in 2012, cuts a mutation out of a gene and replaces it with a gene-piece, a newer type of CRISPR, called base-editing, can repair a mutation…
SMA Plain community carrier screening program yields impactful results
The program identified carriers in the Plain community to deliver innovative treatments presymptomatically to affected newborns
Gene therapy can protect against ALS and SMA-related cell death
Researchers at Karolinska Institutet in Sweden and the University of Milan in Italy have identified a gene in human neurons that protects against the degeneration of motor neurons in the deadly diseases ALS and SMA. Gene therapy in animal models…
Gene therapy/gene editing combo could offer hope for some genetic disorders
Novel approach successfully treated experimental model of rare genetic condition
Gene therapy prevents disorders with alcohol exposure in ALDH2 deficiency
New Rochelle, NY, February 12, 2020–A new study has shown that gene therapy to treat one of the most common hereditary disorders, aldehyde dehydrogenase type 2 (ALDH2) deficiency, may prevent increased risk for esophageal cancer and osteoporosis associated with chronic…
Gene therapy/gene editing combo could offer hope for some genetic disorders
Novel approach successfully treated experimental model of rare genetic condition
Gene therapy prevents disorders with alcohol exposure in ALDH2 deficiency
New Rochelle, NY, February 12, 2020–A new study has shown that gene therapy to treat one of the most common hereditary disorders, aldehyde dehydrogenase type 2 (ALDH2) deficiency, may prevent increased risk for esophageal cancer and osteoporosis associated with chronic…
IDIBELL and Dexeus Mujer will study embryonic development by human embryo genome edition
The team of Dra. Anna Veiga, who leads the study, will optimize the CRISPR/Cas9 technique to modify the human embryo genome
IDIBELL and Dexeus Mujer will study embryonic development by human embryo genome edition
The team of Dra. Anna Veiga, who leads the study, will optimize the CRISPR/Cas9 technique to modify the human embryo genome
Novel techniques for mining patented gene therapies offer promising treatment options
WEST LAFAYETTE, Ind. – The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists from Purdue University and other research institutions around the…
St. Jude’s scientist receives ACGT grant for sarcoma gene therapy
Approach will use genetically engineered immune cells to attack pediatric sarcoma
Novel techniques for mining patented gene therapies offer promising treatment options
WEST LAFAYETTE, Ind. – The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases. Now, a team of scientists from Purdue University and other research institutions around the…
St. Jude’s scientist receives ACGT grant for sarcoma gene therapy
Approach will use genetically engineered immune cells to attack pediatric sarcoma
Rejuvenate Bio launches to help dogs live longer, healthier lives
Combination gene therapy developed at Wyss Institute and Harvard Medical School treats four age-related conditions
Rejuvenate Bio launches to help dogs live longer, healthier lives
Combination gene therapy developed at Wyss Institute and Harvard Medical School treats four age-related conditions
iPS cells to regulate immune rejection upon transplantation
Scientists suggest a new strategy that uses induced pluripotent stem cells (iPSCs) to regulate immune reaction to transplanted tissues. The team, led by Professor Ken-ichiro Seino of Hokkaido University’s Institute for Genetic Medicine, found that thymic epithelium cells derived from…
Tip Sheet: Tracking coronavirus, improving immunotherapies, cancer death rates decline, AAAS meeting and more
Summaries of recent Fred Hutch research studies, plus information on a press event at the upcoming AAAS annual meeting in Seattle.
Fred Hutch scientists host press event on future of medicine at AAAS annual meeting
Fred Hutchinson Cancer Research Center will host a press breakfast Feb. 14 at the annual meeting of the American Association for the Advancement of Science, to be held Feb. 13-16 in Seattle.
UPenn scientists receive ACGT grant to accelerate CAR T-Cell clinical trial
Novel gene therapy approach to prostate cancer already seeing results in clinical trial and may have impact on other solid tumor cancers
ASU scientists boost gene-editing tools to new heights in human stem cells
Proof-of-concept shows genes implicated in Alzheimer’s disease can be accurately edited, with 90 percent efficiency in human stem cells
UPenn scientists receive ACGT grant to accelerate CAR T-Cell clinical trial
Novel gene therapy approach to prostate cancer already seeing results in clinical trial and may have impact on other solid tumor cancers
ASU scientists boost gene-editing tools to new heights in human stem cells
Proof-of-concept shows genes implicated in Alzheimer’s disease can be accurately edited, with 90 percent efficiency in human stem cells
Gene hunting: The power of precision medicine
MU researchers improve animal welfare by discovering genetic mutations that cause disease
Gene hunting: The power of precision medicine
MU researchers improve animal welfare by discovering genetic mutations that cause disease
Organoids (in vitro brains) to study pediatric brain tumors
A large-scale production of in vitro tumors could make it possible to perform large drug screenings to identify new brain cancer drugs
A targeted therapy for triple negative breast cancer may lie existing drugs
Breakthrough findings from a new study point toward the first targeted therapy for an aggressive form of breast cancer that disproportionately affects black women
ASHG survey finds Americans strongly support human genetics research and potential
Respondents want confidence in data privacy, have some knowledge gaps
Organoids (in vitro brains) to study pediatric brain tumors
A large-scale production of in vitro tumors could make it possible to perform large drug screenings to identify new brain cancer drugs
A targeted therapy for triple negative breast cancer may lie existing drugs
Breakthrough findings from a new study point toward the first targeted therapy for an aggressive form of breast cancer that disproportionately affects black women
ASHG survey finds Americans strongly support human genetics research and potential
Respondents want confidence in data privacy, have some knowledge gaps
Six patients with rare blood disease are doing well after gene therapy clinical trial
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.
Tiny Magnetic Structures Enhance Medical Science
Researchers review the state of the art in magnetic nanostructures. One especially interesting advance involves an exotic nanodisc configuration, known as a vortex state, where magnetic moments arrange into a curly geometry.
Gene therapy success in chronic septic granulomatosis
Rare disease of the immune system
Tiny magnetic structures enhance medical science
Researchers review promising new nanomaterials that can be used in cancer diagnosis and as MRI contrast agents
Scientists create listeriosis-immune mice by turning off gene in myeloid cells
The study provides exciting material for understanding the mechanisms of the immune response to pathogenic microorganisms.
Major Asia gene study to help doctors battle disease
An ambitious new study of genes in Asian populations is filling in big gaps in our understanding of human genetics, shedding light on the history of human migration and ultimately aiming to improve our ability to treat disease. Researchers from…