Next-generation gene therapy points to base editing
Base editing has emerged as a new player in the gene therapy arena, with rapid advances in technology and evidence of proof of concept in rare genetic disease and beyond. Read More
'Minibrains' grown from stem cells replicate brain's electrical activity
Researchers have grown 3D human brain tissue from stem cells that replicates the patterns of electrical activity found in the brains of patients with Rett syndrome. These "minibrain organoids" can be used to investigate the underlying causes of neurological disease and screen and test potential drug candidates, according to research published on August 23 in Nature Neuroscience. Read More
McMaster, Sartorius partner on improving biomanufacturing
Sartorius Stedim Biotech and McMaster University, which is located in Hamilton, Ontario, Canada, plan to collaborate on an effort to improve advanced manufacturing processes for antibody and virus-based treatments for diseases like COVID-19 and cancer. Read More
Alternative gene therapies could be effective in treating some rare diseases
Bacteria-mediated genetic transfer has emerged as an alternative gene therapy (AGT) for the treatment of some rare diseases, such as phenylketonuria (PKU). This type of therapy is advantageous because it is easily regulated through established protein expression systems. One company, Synlogic, has developed an AGT candidate that uses a bacterial vector to treat PKU. Read More
Prime editing shows promise for complex diseases like cystic fibrosis
Researchers have demonstrated that prime editing -- a newer version of CRISPR-Cas9 gene editing -- can be successfully employed to correct gene mutations that cause cystic fibrosis. The new study, which tested the technique in human organoids, was published in Life Science Alliance on August 9. Read More
Voyager advances gene therapy platform for CNS diseases
Voyager Therapeutics has launched new gene therapy programs for Huntington's disease, amyotrophic lateral sclerosis caused by mutation in the SOD1 gene, spinal muscular atrophy, and diseases linked to glucosylceramidase beta mutations, powered by its proprietary adeno-associated virus capsids. Read More
Optogenetic gene therapy provides hope for retinitis pigmentosa patients
Optogenetic therapy bypasses traditional methods of repairing defective photoreceptors in the eye by targeting other retinal cells. Recently, three examples of optogenetic therapeutics under development highlight many of the challenges this type of gene therapy faces for the treatment of a devastating genetic eye disease. Read More
Gene therapy tackles 2 rare childhood diseases 2 different ways
Two new studies point to the efficient use of gene therapy to treat deadly childhood genetic diseases. The first study used direct gene therapy infusion to treat a rare neurodevelopmental disorder, while the second study used prenatal base editing in mouse models as proof of concept for the treatment of genetic diseases. These vastly different approaches show the promise of gene therapy for a variety of devastating diseases. Read More
Machine learning helps scientists map the mammalian immune system
The use of advanced machine-learning software has led to the identification of hundreds of new genes that likely affect the working immune system, according to findings from a study published recently in the Proceedings of the National Academy of Sciences of the United States of America. Read More
Do mom's or dad's genes win out in a child's gene expression?
A team of scientists has identified 71 new "imprinted" genes in the mouse genome. The new findings, published in Nature Communications on June 21, help uncover some of the scientific mysteries associated with epigenetics and point to how a parent's genes are uniquely turned off or on in their offspring. Read More
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