Organoid studies in rodents open promising new lines of brain research | Clear explanations of animal research help improve public understanding | Scientists demonstrate safety of Rett syndrome gene therapy in primate study
Two research teams have implanted human brain organoids into lab rodents, and a third team has vascularized human brain organoids, opening new fronts in a line of research that some scientists hope could lead to treatments for stroke, brain injury, autism and other conditions. The rapidly advancing science is also raising complex ethical and regulatory questions that scholars say will need to be explored.
Research institutions, medical research charities, pharmaceutical companies, scientific societies and similar organizations can improve outreach and public understanding of the importance of animal research by including on their websites clear explanations of how animals contribute to medical and scientific advances, as Babraham Institute's website does. Speaking of Research offers free advice and recommendations for improving animal research webpages to provide accurate information about the role of animals in medical, veterinary and scientific research.
A precise dose of an engineered virus containing a properly functioning copy of the MECP2 gene restored motor, learning and memory skills in mouse models of Rett syndrome and extended the animals' life spans. The therapy also produced no adverse effects when tested in cynomolgus monkeys, which in early tests demonstrated more than twofold expression of MECP2 in brain and spinal tissue.
Scientists reported in Nature Biotechnology that they used CRISPR-Cas9 technology to edit the cholesterol-regulating PCSK9 gene in mouse liver cells. The researchers delivered the gene-editing components encased in nanoscale fat particles, and total cholesterol levels in the treated mice dropped by 35%.
Researchers at the Sanford Burnham Prebys Medical Discovery Institute found that DAG, a cyclic peptide with nine amino acids, homed to reactive astrocytes and the neurovascular unit of endothelial cells in mouse models with Alzheimer's disease and also targeted connective tissue growth factor, which is elevated in the brains of both people and mouse models with Alzheimer's prior to the appearance of amyloid plaques. The findings in Nature Communications suggest that DAG may prompt earlier Alzheimer's detection as well as improve drug delivery to affected brain regions in Parkinson's disease, stroke, glioblastoma and brain injuries, researchers said.
An experimental drug corrected an electrical signaling imbalance in the brains of mouse models of autism spectrum disorder, reduced abnormal behaviors and boosted the rodents' performance on cognitive and behavioral tests, researchers reported in Nature Communications. Testing in animals continues, but human trials are also planned.
A veterinary clinic in Southern California and another in Klamath Falls, Ore., are participating in a clinical trial of a stem cell treatment for arthritis in dogs. The clinical trial sponsor hopes to enroll 600 dogs at 20 clinics in the double-blind study, and dogs in the placebo cohort will be eligible for the stem cell treatment at the end of the study, says veterinarian Doug McInnis.
The Focused Ultrasound Foundation is funding three clinical trials on using ultrasound to kill tumors in dogs, and the researchers hope the results will translate to human medical care. Ultrasound is noninvasive and can be used repeatedly, says veterinary program director Kelsie Timbie.
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The Foundation for Biomedical Research (FBR) is the nation’s oldest and largest non-profit dedicated to improving human and animal health by promoting public understanding and support for biomedical research. Our mission is to educate people about the essential role animal research plays in the quest for medical advancements, treatments and cures for both people and animals.