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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

LNP Delivery of Gene-Editing Machinery to Cure Hereditary Tyrosinemia Type 1g

Problem: Hereditary tyrosinemia type 1 (HT1) is a genetic metabolic disease caused by the inability to break down tyrosine, an amino acid present in many meats, dairy products, fruits, and nuts. This disease can be fatal within the first few months of life and increases the risk of liver cancer. The current treatment involves a strict drug regimen and...

Published: 7/10/2024 | Inventor(s): Kiran Musunuru, Madelynn Whittaker, Xiao Wang, William Peranteau, Cara Berkowitz, Ana Dumitru

Keywords(s): Gene Editing, Genetic Disorders, Obesity, Metabolic Disease and Diabetes

Category(s): Technology Classifications > Therapeutics

Novel Mouse Model for Developing Therapeutic Drugs Against Muscle Atrophy

Mouse model stably overexpressing Ant-1 for muscle atrophy therapy development. Background: Muscle disorders are commonly manifested in mitochondria-induced diseases, which are clinically referred to as mitochondrial myopathies. In classic mitochondrial myopathies, caused by mutations in mitochondrial DNA or in nuclear genes encoding mitochondrial...

Published: 7/11/2023 | Inventor(s): Xin Jie Chen, Xiaowen Wang

Keywords(s):

Category(s): Campus > Upstate Medical University, Technology Classifications > Research Tools and Reagents, Technology Classifications > Biomedical Science and Engineering

Synthetic Gene Circuits

Engineering synthetic gene circuits allows scientists to design cells to perform any number of tasks such as biosensing, therapeutic or commodity production or bioremediation to name a few. An important design principle when engineering sophisticated synthetic gene circuits is modularity as it breaks the system down into small modules, thereby...

Published: 2/23/2023 | Inventor(s): Rong Zhang, Hanah Goetz, Juan Melendez-Alvarez, Xiao Wang, Xiaojun Tian

Keywords(s):

Category(s): Computing & Information Technology, Genomic Assays/Reagents/Tools, Life Science (All LS Techs)

A Mutually Inhibitory Network with Positive Autoregulation and Communications (MINPAC)

Biological pattern formation requires complex gene regulation networks and accurate cell-cell coordination. The reaction-diffusion (RD) based clock and wavefront model has been proposed as a mechanism involved in biological pattern formation of repeated and segmented structures. While RD driven pattern formation has been demonstrated in many systems,...

Published: 2/23/2023 | Inventor(s): Fuqing Wu, Samat Bayakhmetov, Changhan He, Qi Zhang, Xingwen Chen, Yang Kuang, Xiao Wang

Keywords(s):

Category(s): Genomic Assays/Reagents/Tools, Life Science (All LS Techs), Computing & Information Technology

Base-Edited Isogenic hPSC Line Generation (BIG-TREE)

Human pluripotent stem cells (hPSCs) are a vital tool not only in research and disease modeling but also in developmental biology and regenerative medicine because of their ability to self-renew and differentiate. Current genome editing tactics in hPSCs require the incorporation of deleterious double-stranded DNA breaks followed by inefficient homology-directed...

Published: 2/23/2023 | Inventor(s): David Brafman, Xiao Wang, Nicholas Brookhouser, Stefan Tekel, Kylie Standage-Beier

Keywords(s):

Category(s): Life Science (All LS Techs), Genomic Assays/Reagents/Tools

Pathogen Molecular Detection Platform

Accurate detection and diagnosis of infectious diseases is paramount to appropriate treatment and better outcomes. Despite tremendous efforts to advance new diagnostic techniques, there are still critical gaps in the healthcare system in quickly diagnosing and responding to infectious diseases. Current diagnostic technologies identify pathogens by detecting...

Published: 2/23/2023 | Inventor(s): Qi Zhang, Xiao Wang, Alexander Green, Duo Ma, Fuqing Wu, Kylie Standage-Beier, Xingwen Chen

Keywords(s):

Category(s): Advanced Materials/Nanotechnology, Applied Technologies, Bioanalytical Assays, Chemistries & Devices, Diagnostic Assays/Devices, Life Science (All LS Techs), Genomic Assays/Reagents/Tools, Proteomic Assays/Reagents/Tools, Medical Diagnostics/Sensors

Targeted Remodeling of Bacterial Genomes Using CRISPR-Nickases

Targeting DNA manipulation is integral to biotechnology and synthetic biology. Large chromosomal rearrangements and deletions have profound impacts on bacterial physiology, such as improved bioproduct levels, increased strain fitness, or changed tolerance to stress and environmental conditions. However, it requires time-consuming and laborious directed...

Published: 2/23/2023 | Inventor(s): Xiao Wang, Kylie Standage-Beier

Keywords(s): Biological Cell Manipulation, Bio-Technology

Category(s): Alternative Energy/Biofuels/Bioplastics/Algae, Genomic Assays/Reagents/Tools, Life Science (All LS Techs)

Model for Gene Expression Tuning and Synthetic Gene Circuit Construction

Genetic circuit engineering has played an important role in many forms of synthetic biology. Based on a large collection of well characterized biological components, complex gene circuits with designed functions can be wired using established biological principles. Currently circuit assembly has two main strategies: one is monocistronic construct, in...

Published: 2/23/2023 | Inventor(s): Fuqing Wu, Xiao Wang, Qi Zhang

Keywords(s):

Category(s): Computing & Information Technology, Genomic Assays/Reagents/Tools, Proteomic Assays/Reagents/Tools, Life Science (All LS Techs)

Novel Cas9 Fusion (iCas9)

In recent years, the true power of predictable and high-fidelity genome engineering for applications in gene therapy and biotechnology is closer than ever to being realized, and the CRISPR and CRISPR-associated (Cas) systems have played an instrumental role in this development. While CRISPR offers great programmability, it isn’t perfect and its...

Published: 2/23/2023 | Inventor(s): Kylie Standage-Beier, Xiao Wang, David Brafman, Nicholas Brookhouser, Parithi Balachandran

Keywords(s):

Category(s): Life Science (All LS Techs), Proteomic Assays/Reagents/Tools, Genomic Assays/Reagents/Tools, Bioanalytical Assays, Chemistries & Devices

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