Search Results - neurodegenerative+diseases

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Overcoming the Blood-Brain Barrier with Peptide-Functionalized Lipid Panoparticles

Peptide-functionalized lipid nanoparticles (pLNPs) for targeted messenger RNA (mRNA) delivery to the brain. Problem: Ionizable lipid nanoparticles (LNPs) have emerged as clinically advanced delivery vehicles for nucleic acids. However, as LNPs typically accumulate to the liver, tissue-specific delivery remains challenging, and systemic LNP delivery...

Published: 12/19/2024 | Inventor(s): Michael Mitchell, Emily Laura Han

Keywords(s): Bioengineering, Drug Delivery, Neurodegenerative Diseases

Category(s): Technology Classifications > Therapeutics

The First Curative Treatment of Phenylketonuria Using Genetic Engineering Approaches

The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects. Problem: Phenylketonuria (PKU) is a severe disorder affecting approximately 1 in 10,000 U.S. newborns. PKU is marked by a genetic mutation in the phenylalanine hydroxylase enzyme (PAH), resulting in phenylalanine (Phe)...

Published: 10/28/2024 | Inventor(s): Kiran Musunuru, Xiao Wang, Dominique Brooks

Keywords(s): Gene Editing, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology

Category(s): Technology Classifications > Therapeutics

Modulating Molecular Chaperone DnaJB6b as a Novel Method to Regulate Tau Seeding and Propagation

Advantages: Semi-high throughput screening accelerates drug discovery by quickly identifying candidates, reducing time to market, and gaining a competitive edge. Molecular Chaperone reduces tau aggregation, preserving cognitive function and improving patient quality. Employs a precision medicine approach targeting tauopathy mechanisms, potentially...

Published: 9/11/2024 | Inventor(s): Abigail Esquivel, Laura Blair

Keywords(s): Alzheimer's Disease, Neurodegenerative Diseases

Category(s): Technology Classifications > Medical > Neuroscience, Technology Classifications > Medical > Biotechnology, Technology Classifications > Medical > Therapeutics

An Adaptive, Language-Agnostic, and Generalizable Method to Determine the Hearing Level and Speech Perception Abilities of a Patient

This hearing test is tuned to a patient’s hearing level and changes based on performance to accurately diagnose hearing ability Problem: Currently, no single hearing evaluation can determine multiple facets of an individual’s hearing ability. For example, some tests may determine hearing awareness at specific frequencies, but gather no data...

Published: 8/12/2024 | Inventor(s): Jason Brant, Tiffany Hwa

Keywords(s): Aging, Neurodegenerative Diseases, Software, Surgery

Category(s): Technology Classifications > Diagnostics, Technology Classifications > Testing & Measurement

Novel Helical Peptodomimetics that Prevent the Formation of Aβ Oligomerization in Alzheimer’s Disease (AD)

Advantages: Developed sulfonyl-γ-AA peptide helical peptidomimetics that can prevent the formation of neurotoxic Aβ oligomerization. High Specificity: The lead sulfonyl-γ-AApeptide helical foldamer, Ab-6, exhibits precise targeting to the central region of Aβ42 and induces α-helix conformation in Aβ. Sequence-Specific...

Published: 11/6/2024 | Inventor(s): Jianfeng Cai, Heng Liu

Keywords(s): Alzheimer's Disease, Biotechnology, Neurodegenerative Diseases

Category(s): Technology Classifications > Medical > Biomedical Engineering, Technology Classifications > Medical > Biotechnology, Technology Classifications > Medical > Neuroscience

New Device Material for a Flexible Chronic Implantable Neural Interface

Advantages: Superior biocompatibility reduces immune responses. Excellent electrical conductivity enhances signal transmission. Exceptional mechanical strength enables durable and flexible probes. Summary: Implantable neural interfaces (INIs) are devices configured to connect with the human nervous system through electrical means. Such...

Published: 9/3/2024 | Inventor(s): Stephen Saddow, Chenyin Feng, Christopher Frewin

Keywords(s): Drug Delivery and Nanomedicine, Medical Therapeutics Cell Therapy, Nanotechnology, Neurodegenerative Diseases

Category(s): Technology Classifications > Medical > Biomedical Engineering, Technology Classifications > Medical > Biomaterials, Technology Classifications > Medical > Medical Devices, Technology Classifications > Medical > Neuroscience

Silencing of PRLΔE1 expression: a gene-agnostic treatment for retinal degenerations

Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival. Problem: Age-related macular degeneration (AMD) and inherited retinal degeneration (IRDs) have extreme mutational heterogeneity, and many IRDs are orphan diseases. Developing individual gene therapies for each causative gene is impractical...

Published: 7/2/2024 | Inventor(s): William Beltran, Raghavi Sudharsan

Keywords(s): Drug Target, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Ophthalmology, Orphan Disease

Category(s): Technology Classifications > Therapeutics

PET Radiotracers for Diagnosis and Patient Stratification of Neurodegenerative Disorders

Small molecules that bind to alpha-synuclein deposition and can be used for PET imagining in patients with synucleinopathies Problem: Imaging alpha-synuclein (αS) using noninvasive techniques such as positron emission tomography (PET) can help with the early diagnosis of synucleinopathies. Previous PET radiotracers suffer from low binding affinity...

Published: 6/6/2024 | Inventor(s): Robert Mach, E. James Petersson

Keywords(s): Imaging Agents, Neurodegenerative Diseases, Radiology, Small Molecule

Category(s): Technology Classifications > Chemical Processes and Synthesis, Technology Classifications > Diagnostics, Technology Classifications > Imaging, Technology Classifications > Research Tools & Reagents

Secreted UBE3A for the Treatment of Neurological Disorders

Advantages: Novel vector, composition, and method of treating a neurological disorder with UBE3A deficiency. High fidelity to human Angelman syndrome. Facilitates comprehensive symptom representation for diverse treatment investigations. Allows innovative experiments for pioneering treatment development. Summary: Angelman...

Published: 1/2/2024 | Inventor(s): Kevin Nash

Keywords(s): Animal Models, Medical Therapeutics Gene Therapy, Neurodegenerative Diseases, Neuroscience

Category(s): Technology Classifications > Medical > Biomedical Engineering, Technology Classifications > Medical > Neuroscience

Direct and Effective Method To Reverse The Effects of Fragile X Syndrome

A method of treating Fragile X syndrome by correcting the abnormal DNA clusters causing the characteristic symptoms. Problem: Fragile X syndrome (FXS) affects approximately 1 in 4,000 males and 1 in 8,000 females and is characterized by symptoms such as: Inherited Intellectual Disability Anxiety Attention Deficit Disorder (ADD) Skin, Ovaries...

Published: 3/8/2024 | Inventor(s): Jennifer Phillips-Cremins, Linda Zhou, Chunmin GE

Keywords(s): Bioengineering, Gene Therapy, Genetic Disorders, Neurodegenerative Diseases, Neurology

Category(s): Technology Classifications > Therapeutics, Technology Classifications > Vaccines

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