China is helping to advance gene and cell therapy and genome editing research and clinical development by creating novel viral and nonviral vectors for gene delivery and innovative applications of CRISPR technology in a broad range of disease areas.
Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today in the New England Journal of Medicine. The study demonstrates the impact the drug, nusinersen, can have on older patients with this progressive neuromuscular disorder.
A new method pioneered by UCLA researchers enables an average biochemistry laboratory to make its own gene sequences for only about $2 per gene, a process that previously would require its researchers to pay a commercial vendor $50 to $100 per gene. The approach, described in the journal Science, will make it possible for scientists to mass produce thousands of genes screen for their roles in diseases.
In one of the first successes of its kind, researchers have inhibited the spreading of cancer cells from one part of the body to another. In doing so, they relied on a new model of how cancer metastasizes that emphasizes epigenetics, which examines how genes are turned on and off.
Employing CRISPR/Cas9 advancements, UC San Diego researchers are using new active genetics technology to reveal new fundamental mechanisms that control gene activity. The authors also provide experimental validation for using active genetics as an efficient means for targeted gene insertion, or 'transgenesis,' and single-step replacement of genetic control elements.
Gene therapy can potentially correct genetic disorders by directly editing defective genes. CRISPR-Cas9 is a popular gene-editing technology whose clinical utility is limited by its tendency to produce unintended genetic errors. Researchers centered at Osaka University developed a modified CRISPR-Cas9 system that uses single-stranded nicking, rather than DNA cleavage, to generate highly precise changes to a target gene. The technique offers a more accurate and safer editing strategy for future gene therapy applications.
Houston Methodist researchers have a new explanation for what causes the lungs' airways to close during asthma attacks that could change the lives of the 300 million people worldwide who suffer from asthma. The discovery holds promise for developing a new class of drugs that is radically different from the steroids currently used to treat it. The NIH-funded study is in the Feb. 5 issue of the Journal of Experimental Medicine.
Scientists from the University of Zurich have redesigned an adenovirus for use in cancer therapy. To achieve this they developed a new protein shield that hides the virus and protects it from being eliminated. Adapters on the surface of the virus enable the reconstructed virus to specifically infect tumor cells.
Updated results from a global clinical trial of the CAR T-cell therapy, tisagenlecleucel, a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL), reveal that children and young adults continued to show high rates of durable, complete remission of their disease. Most side effects were short-lived and reversible.
Researchers have described severe, life-threatening toxicity in monkeys and piglets given high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector capable of accessing spinal cord neurons.