May 2, 2024

Scientists Discover New Promising Target for Alzheimer’s Disease Treatment

Advertisement is the most typical reason for dementia in older adults, with an estimated 6 million Americans presently living with the illness.
It is a progressive brain disorder that gradually damages memory and thinking abilities. Foundational research at Penn Medicine led by Virginia M.Y. Lee, Ph.D., the John H. Ware III Professor in Alzheimers Research in Pathology and Laboratory Medicine, and the late John Q. Trojanowski, MD, Ph.D., a previous teacher of Geriatric Medicine and Gerontology in Pathology and Laboratory Medicine, exposes that a person of the underlying reasons for neurodegenerative diseases is neurofibrillary tangles (NFTs) of tau proteins, which trigger the death of nerve cells, resulting in the symptoms of AD, like loss of memory.
Individuals with Alzheimers disease (ADVERTISEMENT) have actually increased neurofibrillary tangles (NFTs) of tau proteins however decreased TRIM11, as revealed in the bottom 2 quadrants, compared to individuals without AD, displayed in the top 2 quadrants. Credit: Penn Medicine.
In addition to advertisement, aggregation of tau proteins into NFTs is related to over 20 other dementias and movement conditions consisting of progressive supranuclear palsy, Picks disease, and persistent terrible encephalopathy, collectively called tauopathies. How and why tau proteins clump together and form the fibrillar aggregates that makeup NFTs in clients with these diseases remains unclear. This significant gap in understanding has actually made the advancement of effective therapies challenging for scientists.
” Most organisms have protein quality assurance systems that eliminate defective proteins and prevent the misfolding and accumulation of tangles– like the ones we see with tau proteins in the brain of those with tauopathies– but previously we didnt understand how this operates in human beings, or why it malfunctions in some individuals and not others,” said senior author, Xiaolu Yang, Ph.D., a professor of Cancer Biology at Penn. “For the very first time, we have recognized the gene that supervises tau function, and have a promising target for establishing treatments to prevent and slow the development of Alzheimers illness and other associated disorders.”
Yang and his group, consisting of very first author Zi-Yang Zhang, Ph.D., a postdoctoral researcher in Yangs laboratory, previously found that TRIM proteins play a crucial function in protein quality control in animal cells. After analyzing over 70 human TRIMs, they found that TRIM11 has a major function in suppressing tau aggregation. TRIM11 has 3 main functions connected to the quality control of tau proteins. First, it binds to tau proteins, particularly the mutant versions that trigger illness, and assists remove them. Second, it serves as a “chaperone” for tau, avoiding the proteins from misfolding. TRIM11 dissolves pre-existing tau aggregates.
Utilizing postmortem brain tissues of 23 people with AD and 14 health controls from the Center for Neurodegenerative Disease Research tissue bank– produced and maintained by Lee and Trojanowski– scientists validated these findings and found that levels of TRIM11 protein are considerably reduced in the brains of people with advertisement, compared to healthy control people.
To figure out the prospective utility of TRIM11 as a restorative agent, scientists utilized adeno-associated viral vector (AAV), a tool typically utilized in gene treatment, to deliver the TRIM11 gene into the brain of several mouse models. Scientists found that mice with tau pathologies getting the TRIM11 gene showed a significant decline in the advancement and accumulation of NFTs, and had much enhanced cognitive and motor capabilities.
” Not just do these findings inform us that TRIM11 might play a crucial role in securing individuals from Alzheimers and comparable illness, however we likewise see that we may be able to develop future treatments that renew TRIM11 in individuals with lower levels,” said Yang. “We aspire to deal with our associates to check out the possibility of developing gene therapies that stop the development of neurodegenerative disease.”
Reference: “TRIM11 secures versus tauopathies and is down-regulated in Alzheimers disease” by Zi-Yang Zhang, Dilshan S. Harischandra, Ruifang Wang, Shivani Ghaisas, Janet Y. Zhao, Thomas P. McMonagle, Guixin Zhu, Kenzo D. Lacuarta, Jianing Song, John Q. Trojanowski, Hong Xu, Virginia M.-Y. Lee and Xiaolu Yang, 28 July 2023, Science.DOI: 10.1126/ science.add6696.
This research study was supported by the National Institutes of Health (R01CA243520, UL1TR000003) and funding received by Penn under a sponsored research study contract with Wealth Strategy Holding Limited.
Yang is an inventor on patents and patent applications owned by the University of Pennsylvania associated to TRIM proteins, and is a co-founder and equity holder of Evergreen Therapeutics LLC, which got investments from Wealth Strategy Holding Limited. Penn and Yang have actually either gotten, or may receive in the future, monetary factor to consider associated to the licensing of specific Penn intellectual home to Evergreen Therapeutics LLC.

A new study found that the protein TRIM11 can hinder neurodegeneration and enhance cognitive and motor functions in animal designs comparable to Alzheimers. The research study recommends TRIM11 plays a considerable function in eliminating harmful protein tangles related to neurodegenerative illness.
Levels of the TRIM11 protein were discovered to be reduced in models of Alzheimers illness, according to new research from Penn Medicine The study recommends that restoring the protein might enhance cognitive and motor function.
New research from the Perelman School of Medicine at the University of Pennsylvania has actually found that a gene encoding a protein linked to tau production– tripartite concept protein 11 (TRIM11)– was found to reduce deterioration in small animal designs of neurodegenerative diseases comparable to Alzheimers illness (AD), while improving cognitive and motor capabilities.
In addition, the study determined TRIM11 as a considerable gamer in the elimination of protein tangles that cause neurodegenerative diseases such as advertisement. The findings were recently released in the journal Science.

In addition to AD, aggregation of tau proteins into NFTs is associated with over 20 other dementias and motion disorders including progressive supranuclear palsy, Picks illness, and persistent distressing encephalopathy, jointly understood as tauopathies. How and why tau proteins clump together and form the fibrillar aggregates that makeup NFTs in patients with these diseases stays uncertain. Yang and his team, consisting of first author Zi-Yang Zhang, Ph.D., a postdoctoral scientist in Yangs lab, formerly discovered that TRIM proteins play an essential function in protein quality control in animal cells. TRIM11 has three main functions related to the quality control of tau proteins. It binds to tau proteins, particularly the mutant variations that cause illness, and assists eliminate them.