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Search Results - gene+editing
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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
Strategies for Mitigating Acute Side Effects of Lung-Targeted Nanocarriers
Approaches and Design Techniques to Alleviate Coagulation-Related Acute Side Effects of Lung-Targeted Nanocarriers Problem: Lipid nanoparticles (LNPs) play a crucial role in drug delivery, enabling targeted drug administration to specific tissues. LNPs tailored for lung-specific delivery often incorporate cationic (positively charged) lipids. Unfortunately,...
Published: 6/26/2024
|
Inventor(s):
Jacob Brenner
,
Vladimir Muzykantov
,
Jacob Myerson
,
Oscar Marcos Contreras
,
Marco Zamora
,
Serena Omo-Lamai
Keywords(s):
Bioengineering
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Nanotechnology
,
Platform Technology
,
Pulmonary
Category(s):
Technology Classifications > Therapeutics
RGD Peptide-Based Lipids For Targeted mRNA Delivery and
Gene Editing
Applications
A combinatorial library of RGD peptide (arginine-glycine-aspartic acid)-based lipids designed to formulate lipid nanoparticles for targeted mRNA delivery to the liver and the spleen. Problem: Lipid Nanoparticles (LNPs) are becoming an established choice of vehicle to deliver mRNA-based therapeutics, including vaccines, protein replacement therapies,...
Published: 2/7/2024
|
Inventor(s):
Michael Mitchell
,
Lulu Xue
,
Jingya Qin
Keywords(s):
Bioengineering
,
Drug Delivery
,
Gene Editing
,
Oncology
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
,
Technology Classifications > Vaccines
Cholesterol Analogues Incorporated Into LNPs For Enhanced mRNA Delivery
Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells. Problem: In recent times, the delivery of mRNA has become a major focus for therapeutics. Intracellular delivery is the major challenge. Intracellular delivery requires ionizable lipids for mRNA delivery. In addition to this, these constituents...
Published: 2/6/2024
|
Inventor(s):
Michael Mitchell
,
Margaret Billingsley
,
Savan Patel
,
Xuexiang Han
,
Hanwen Zhang
Keywords(s):
Bioengineering
,
Cell Therapy
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Immunoncology
,
Immunotherapy
,
Inflammation
,
Nanotechnology
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
Amniotic Fluid Stabilized Lipid Nanoparticles (LNPs) for In Utero Intra-Amniotic mRNA Delivery
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac Problem: Many prenatal genetic diseases suffer from ineffective postnatal treatments. For this reason, therapies that are delivered to the fetus before birth hold promise to treat diseases prior to or in the early stages of...
Published: 2/7/2024
|
Inventor(s):
Michael Mitchell
,
Kelsey Swingle
,
Margaret Billingsley
,
William Peranteau
Keywords(s):
Bioengineering
,
Cell Therapy
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Nanotechnology
,
Orphan Disease
,
Platform Technology
Category(s):
Technology Classifications > Therapeutics
Ligand-Tethered Lipid Nanoparticles for Targeted RNA Delivery to Treat Liver Fibrosis
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver. Problem: Lipid nanoparticles (LNPs) have been successfully used in FDA-approved therapies to deliver messenger RNA (mRNA) and small interfering RNA (siRNA) . One of the biggest challenge in developing...
Published: 2/7/2024
|
Inventor(s):
Michael Mitchell
,
Xuexiang Han
Keywords(s):
Bioengineering
,
Drug Delivery
,
Gene Editing
,
Gene Therapy
,
Genetic Disorders
,
Immunology
,
Immunoncology
,
Platform Technology
,
Regenerative Medicine
Category(s):
Technology Classifications > Therapeutics
,
Technology Classifications > Vaccines
ADENO-ASSOCIATED VIRUS (AAV) DIRECTED ANTIOXIDATIVE GENE THERAPY FOR THE PREVENTION AND TREATMENT OF NOISE-INDUCED HEARING LOSS
SOD1 gene therapy (blue) shows a persistent neuroprotective effect compared to the severe hearing loss observed in the control animals (purple) Invention Summary: Hearing loss is the third most common chronic health problem in the United States, with noise-induced hearing loss effecting nearly 1 out of every 4 adults (over 60M). Inner ear hearing...
Published: 7/16/2024
|
Inventor(s):
P. Ashley Wackym
,
Todd Mowery
Keywords(s):
Gene editing
Category(s):
Technology Classifications > Healthcare & Life Sciences
Controlled Muscle-Specific Gene Delivery
Treatment with the AAV gene therapy with doxycycline (B) induced expression of the target gene. Invention Summary: Adeno-associated virus (AAV) vector-based gene therapy has the potential to treat a number of rare diseases. However, only two AAV-based gene therapies have been approved by the FDA, primarily due to toxicity and lack of specificity. Researchers...
Published: 5/2/2024
|
Inventor(s):
Renping Zhou
Keywords(s):
Gene editing
Category(s):
Technology Classifications > Gene Therapy
,
Technology Classifications > Healthcare & Life Sciences
Novel gene drive based on eliciting piRNA biogenesis in insect pests
Transgenic Anopheles gambiae larvae expressing fluorescent marker gene Invention Summary: Malaria is one of the most impactful diseases in tropical and subtropical regions and is transmitted through human contact with infectious mosquitoes. Crop pests create a large economic and social impact through direct damage to plants and spread...
Published: 6/18/2024
|
Inventor(s):
Cory Henderson
,
Christina Bergey
Keywords(s):
Gene editing
Category(s):
Technology Classifications > Healthcare & Life Sciences
,
Technology Classifications > Pest Control
,
Technology Classifications > Ag Biotech
Novel Maturation‐defective Attenuated Chlamydia Vaccine
Invention Summary: Millions of American men and women are infected with Chlamydia each year. Without proper treatment, a substantial proportion of chlamydia-infected women may develop infertility and other health problems. To date, no Chlamydia vaccine is available. Chlamydia is an obligate intracellular bacterium with a unique...
Published: 5/2/2024
|
Inventor(s):
Huizhou Fan
Keywords(s):
Gene editing
Category(s):
Technology Classifications > Vaccine
,
Technology Classifications > Healthcare & Life Sciences
,
Technology Classifications > Infectious Disease
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