27 April 2024
Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
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Mosaics of predisposition cause skin disease
Clarifying the cause of a skin disease led to the discovery of a new disease-causing gene, a new category of diseases, and new perspectives for both counseling and therapy. The discovery is the first time that epigenetic silencing, the 'switching off' of an otherwise intact gene, has been recognized as the cause for a skin disease.
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Nanoparticle delivery of FZD4 to lung endothelial cells inhibits lung cancer progression and metastases
A recent breakthrough study has shown potential to improve therapeutic outcomes for patients suffering from lung cancers.
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New insight into combating drug-resistant prostate cancer
New research sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the activity of coregulator proteins.
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An hereditary liver disease cured with the help of gene scissors
Researchers have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on cells.
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An immunotherapy to overcome resistant leukemia
Researchers have discovered that a specific mutation in the cancer cells of an aggressive type of blood cancer can prevent novel immunotherapies such as CAR T-cell therapy from working. Their study also explains why the cancer cells are resistant and how this resistance can be overcome: through concomitant pharmacotherapy or genetically improved CAR T-cells.
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Gene discovered that can protect against severe muscle disease
A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular dystrophies.
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Researchers closing in on genetic treatments for hereditary lung disease, vision loss
Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy for hereditary conditions like cystic fibrosis and inherited vision loss.
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A better way to deliver fetal therapy for serious genetic disorders
In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as effective as delivering it to the fetal brain via cerebrospinal fluid. The experiment was done in mice with a genetic disorder called Angelman syndrome.
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Genetic signature may predict response to immunotherapy for non-small cell lung cancer
A new study identified a set of 140 genes that may help predict enhanced disease-free survival in patients with non-small cell lung cancer (NSCLC) treated with a combination of immunotherapy and low-dose radiation. The results suggested that this 'gene signature' could be used to identify a subclass of lung tumors that is more likely to be eradicated by immunotherapies.
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Gene-editing offers hope for people with hereditary disorder
A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.
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Researchers identify key characteristics associated with improved CAR T outcomes in large B cell lymphoma
Axi-cel CAR T targets the CD19 molecule on large B-cell lymphoma cells. The ZUMA-7 trial demonstrated that axi-cel reduced the risk of disease progression, the need for new therapy, or death by 60% compared to standard therapy. Despite these positive outcomes in event-free survival and overall survival, some patients did not respond well to therapy or relapsed quickly after treatment. Researchers wanted to assess if there were specific tumor characteristics associated with improved outcomes that could better inform treatment selection.
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A novel switch to turn genes on/off on cue, a promising step toward safer gene therapy
Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect. Researchers now report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications.
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Mice possess natural gene therapy system
A previously mysterious small RNA molecule in mice is found to play a crucial role in gene expression, and may be the first identified member of a new class of regulatory RNAs.
