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Method for using alternative splicing to control specificity of gene therapy

Unmet NeedViral-based gene therapy holds tremendous promise for the treatment of human diseases. Using genetically engineered carriers called vectors to deliver genes into cells, gene therapy can help replace a mutated gene with a healthy copy or even introduce a new gene into the body to fight a disease. However, an important concern when designing...

Published: 5/9/2024 | Inventor(s): Jonathan Ling, Seth Blackshaw

Keywords(s): Drug Delivery Mechanism, Gene Therapy, Single, Therapeutic Matter, Therapeutics, Therapy Type

Category(s): Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms, Technology Classifications > Research Tools > Vectors & Plasmids

Targeted capture of custom nucleotide sequences for single-cell sequencing

Value Proposition· Implementation only requires simple modification of current capture beads.· Allows for targeting of custom sequences of interest that may be cell-type specific or expressed at low levels.· Targets RNA molecules without polyA tails.· Inexpensive modification to existing commercial methods. Technology DescriptionResearchers...

Published: 5/14/2024 | Inventor(s): Jonathan Ling, Katherine Irwin, Irika Sinha, Philip Wong, Linda Orzolek, Tyler Creamer

Keywords(s):

Category(s): Technology Classifications > Research Tools > Nucleic Acids, Technology Classifications > Research Tools > Vectors & Plasmids, Technology Classifications > Research Tools > Assays

Identification of TDP-43 Cryptic Exons as Functional Fluid Biomarkers for Alzheimer's Disease and Related Dementia

JHU C16735Unmet Need: TDP-43 (Tar DNA-binding protein 43) acts as a guardian of the transcriptome by repressing the splicing of non-conserved, unannotated cryptic exons. Loss of TDP-43 function causes neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Limbic-Predominant Age-Related TDP-43 Encephalopathy...

Published: 5/13/2024 | Inventor(s): Philip Wong, Jonathan Ling

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities > Proteins, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Dementia

Local Sustained-release Nanocrystal Formulations to Treat and Prevent GI Fibrosis

Unmet Need: Gastrointestinal (GI) fibrosis, the accumulation of scar tissue in the intestinal wall as a complication in common GI autoimmune diseases such as ulcerative colitis (UC) and Crohn’s disease (CD), leads to intestinal strictures, abdominal pain, and reduced intestinal motility. Current pharmacological and surgical treatment methods have significant...

Published: 5/10/2024 | Inventor(s): Laura Ensign-Hodges, Florin Selaru, Min Kyung Joo, Rachel Shapiro, Ling Li

Keywords(s):

Category(s): Clinical and Disease Specializations > Gastroenterology

New use of Sulconazole to Treat and Prevent GI Fibrosis

Published: 5/10/2024 | Inventor(s): Florin Selaru, Laura Ensign-Hodges, Ling Li, Min Kyung Joo

Keywords(s):

Category(s): Clinical and Disease Specializations > Gastroenterology

Locoregional Chemotherapy for Liver Cancers

Unmet NeedOver 800,000 people are diagnosed with liver cancer annually. As the leading cause of cancer death worldwide, liver cancer accounts for more than 700,000 deaths annually. Recently, Johns Hopkins researchers have developed new chemotherapeutic drugs to treat liver cancer that demonstrate higher efficacy than the current standard of care for...

Published: 5/9/2024 | Inventor(s): Florin Selaru, Ling Li

Keywords(s): Antagonists/Inhibitors, Cancers, Disease Indication, Drug Delivery Mechanism, Liver Cancer, Non-novel, Predicted Novelty, Repurposed, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Technology Classifications > Therapeutic Modalities > Repurposed Drugs, Technology Classifications > Therapeutic Modalities > Small Molecules, Clinical and Disease Specializations > Oncology > Liver Cancer

Liver-directed gene delivery via endoscopic retrograde cholangiopoancreatograpy (ERCP) directed hydrodynamic injection

Unmet NeedGene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of gene therapy trials has been steadily increasing in the United States, with around 170 trials taking place in 2015. All of these trials aim to treat the causes of different diseases instead of only relieving symptoms. Many...

Published: 5/9/2024 | Inventor(s): Florin Selaru, Vivek Kumbhari, Ling Li

Keywords(s): Drug Delivery Mechanism, Therapeutic Matter, Therapeutics

Category(s): Technology Classifications > Therapeutic Modalities > Therapeutic Delivery Platforms

Target Validation of Splicing Repression, a Major Function of TDP-43 in the Motor Neuron

Unmet NeedAmyotrophic lateral sclerosis (ALS) is a rare but debilitating disease characterized by the death of motor neurons that eventually leads to difficulty moving, speaking, swallowing, and breathing. The fundamental cause of ALS is unknown and as a result treatment options are primarily restricted to physical therapy to help prolong muscle strength....

Published: 5/9/2024 | Inventor(s): Philip Wong, Aneesh Donde, Jonathan Ling, Liam Chen, Mingkuan Sun

Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets

Mini-cancers Utilization as Decision Tools for Personalized Cancer Drug Regimens

The present invention includes methods for identifying chemotherapy treatments specifically suited for each cancer patient. The methods include obtaining a biopsy from the cancer patient; establishing a cancer culture from the biopsy; mixing the cancer culture with a matrix forming mini cancers. The mini cancers are then used to screen chemotherapy...

Published: 5/9/2024 | Inventor(s): Florin Selaru, Ling Li

Keywords(s): Assay, Cancers, Clinical Diagnostics, Discovery/Research Tools, Disease Indication, In Vitro Diagnostics, In Vitro Research Tool, Research Instrument, Target-based Screening Tool

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Oncology, Technology Classifications > Diagnostics > Companion Diagnostics, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Technology Classifications > Research Tools

TDP-43 Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative Diseases

C13621: Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative DiseasesAbstractA novel mechanism underlying the suppression of non conserved cryptic exons. This mechanism is essential to maintain intron integrity, and is compromised in ALS-FTD, indicating that loss of function underlies pathogenesis of disease. Publication...

Published: 5/9/2024 | Inventor(s): Philip Wong, Jonathan Ling

Keywords(s): Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Basic Research Biomarker, CNS and Neurological Disorders, Dementia, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock Out Mouse Model, Mouse Model

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools > Animal Models, Technology Classifications > Research Tools, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia

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