Press release
Wednesday May 21, 2025
A breakthrough funded by NIH could allow targeted therapies for many neurological disorders.
Research teams funded by the National Institutes of Health (NIH) have created a versatile set of gene delivery systems that can reach different types of neural cells in the human brain and spinal cord with exceptional precision. These administration systems are an important step towards precise gene therapy in the brain which could safely control the wandering brain activity with great precision. On the other hand, current therapies of brain disorders mainly deal with symptoms.
The new delivery systems transport genetic material in the brain and spinal cord for use targeted by specific cells of cells. This platform has the potential to transform the way scientists can study neural circuits. It provides researchers with gene administration systems for various species used in research, without the need for genetically modified or transgenic animals. Examples include the illumination of fine brain cell structures with fluorescent proteins and activation or silence of circuits that control behavior and cognition.
“Imagine this new platform as a delivery truck depositing genetic plans specialized in specific cellular districts in the brain and spinal cord,” said John Ngai, director of NIH Brain research by advancing innovative neurotechnologies® Initiative, or the brain initiative®. “With these delivery systems, we can now access and manipulate specific cells in the brain and spinal cord – access that was not possible before this scale.”
The new delivery tools, which use a small adeno-associated virus (AAV) stripped of DNA to target cells, can be widely applied through many species and experimental systems, including small samples of tissue eliminated during human brain surgeries. The delivery systems have been tested or validated in intact life systems, which is an important step to introduce new tools for general use. The newly published toolbox includes:
- Dozens of delivery systems that selectively target the types of key brain cells, in particular excitatory neurons, inhibitory interneurons, striatal and cortical subtypes, brain blood cells and difficult to buy neurons in the spinal cord that controls the body movement and are damaged in several neurological diseases, Amyotrophic lateral sclerosis (ALS) and spinal bodybuilding, amyotrophy atrophy
- IT programs fueled by artificial intelligence (AI) which can identify genetic “light switches”, known as activators, which activate genes in specific brain cells, using data from many different species – reducing considerable time and efforts for scientists in search of these genetic switches.
Overall, this collection of research tools will considerably accelerate understanding of the human brain. Above all, the toolbox provides access to specific brain cells in the prefrontal cortex, an area that is essential for decision -making and human traits only. With other tools from the collection, scientists can better study individual cells and communication routes known to be affected in several neurological diseases. These include crisis disorders, ALS, Parkinson’s disease, Alzheimer’s disease and Huntington’s disease – as well as various neuropsychiatric conditions.
AAV -based treatments are already approved for certain conditions, such as spinal muscular atrophy for which an approval in 2016 of gene therapy known as Zolgensma transformed the life of infants and young children who faced severe disability or early death. The new collection of gene resources of genes throws the basics of more precise treatments that only target cells affected in the brain, spinal cord or brain blood vessels.
The toolbox is available in distribution centers, including Addgene, a global provider of genetic research tools. This collection of publications offers researchers standard operational procedures and user guides for these tools.
The work is supported by the NIH Brain research by advancing innovative neurotechnologies® Initiative, or the brain initiative®. The funding issued less than four years ago has launched a large -scale project and managed by team to design new molecular tools which can be useful for many research laboratories. Armmentarium for access to precision cerebral cells aims to develop precise and reproducible access to cells and circuits in experimental research models of the brain and spinal cord. The large -scale project brings together experts in the field of molecular biology, neuroscience and artificial intelligence (AI). The eight articles appear in the May 21 issue of reviews Neuron,, Cells, cellular reports, cell genomics and cellular relationship methods.
Grants: UF1MH130701, UH3MH120096, U24MH133236, UF1MH128339, UM1MH30981, R01MH123620, U19MH1114830, P5101010425, U420D01123 UH3MH120094 UF1MH130881, F30DA053020, R01FD007478, U01AG076791, R35GM127102, RF1MH114126, UH3MH120095, RF1M121274, R01MH113005, UH3MH120095, R01MH113005, UH3MH1200950, R01MH113005, UH3MH1200950
Brain research by progressing innovative neurotechnologies® Initiative and The Brain Initiative® are registered brands of the American Department of Health and Social Services.
The NIH Brain Initiative, a multidisciplinary collaboration in 10 NIH institutes and centers, is only positioned for cross -discoveries in neuroscience to revolutionize our understanding of the human brain. By accelerating the development and application of innovative neurotechnologies, the Brain Initiative® allows researchers to understand the brain at unprecedented retail levels in health and disease, let’s improve the way we treat, prevent and healing brain disorders. The Brain initiative involves a multidisciplinary network of federal and non -federal partners whose current research missions and research portfolios complete the objectives of the brain initiative.
On the National Institutes of Health (NIH):
The NIH, the country’s medical research agency, includes 27 institutes and centers and is a component of the American department of health and social services. NIH is the main federal agency that leads and supports basic, clinical and translational medical research, and studies the causes, treatments and remedies for common and rare diseases. For more information on the NIH and its programs, visit www.nih.gov.
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