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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Lipid-Lowering Drugs: Statins and Miscellaneous Agents01:20

Lipid-Lowering Drugs: Statins and Miscellaneous Agents

Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
Lipid Absorption01:24

Lipid Absorption

Dietary triglycerides from chyme in the duodenum are mixed with bile salts produced by the liver to emulsify fats. As a result, large droplets are broken down into smaller ones, increasing the surface area for enzymatic action. Once emulsified, pancreatic lipases hydrolyze the triglycerides into free fatty acids and monoglycerides.
These breakdown products bind with bile salts and lecithin to form micelles, which quickly pass between microvilli to come in close contact with the apical...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Cholesterol: Significance and Regulation01:29

Cholesterol: Significance and Regulation

Although not a source of energy, cholesterol plays a significant role as a foundational structure for bile salts, steroid hormones, and vitamin D, as well as being a crucial component of plasma membranes. Approximately 15% of blood cholesterol is derived from our diet, with the remainder synthesized from acetyl CoA by the liver and intestines. Cholesterol is eliminated from the body through its conversion into bile salts, which are eventually discarded in the feces.
Considering cholesterol and...
Lipid-derived Compounds in the Human Body01:31

Lipid-derived Compounds in the Human Body

Fats and lipids are crucial components in the human body. Some lipid-derived compounds, such as fat-soluble vitamins, eicosanoids, lipoproteins, and glycolipids, also play unique roles to support various  biological processes .
Fat-soluble Vitamins
Fat-soluble vitamins, including vitamins A, D, E, and K, are required in minimal quantities, but their deficiencies can lead to severely abnormal physiological conditions. For example, vitamin A deficiency can cause night blindness, dry skin, delayed...

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Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
08:29

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

Published on: February 1, 2019

Novel cholesterol-based cationic lipids for gene delivery.

Darya A Medvedeva1, Mikhail A Maslov, Roman N Serikov

  • 1Institute of Chemical Biology and Fundamental Medicine, SB RAS, 8 Lavrentiev Avenue, Novosibirsk 630090, Russian Federation.

Journal of Medicinal Chemistry
|October 15, 2009
PubMed
Summary

New biodegradable cholesterol-based lipids show promise for gene therapy. These lipids efficiently deliver nucleic acids into cells with low toxicity, advancing gene delivery strategies.

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Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy
15:55

Long-term Silencing of Intersectin-1s in Mouse Lungs by Repeated Delivery of a Specific siRNA via Cationic Liposomes. Evaluation of Knockdown Effects by Electron Microscopy

Published on: June 21, 2013

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Nanotechnology

Background:

  • Gene therapy offers a promising approach for treating human diseases.
  • Effective gene delivery vehicles are crucial for successful gene therapy.
  • Developing safe and efficient non-viral gene delivery systems is an ongoing challenge.

Purpose of the Study:

  • To synthesize and characterize novel biodegradable cholesterol-based cationic lipids.
  • To investigate the structure-activity relationships of these lipids in gene delivery.
  • To identify optimal lipid formulations for enhanced nucleic acid delivery and low cellular toxicity.

Main Methods:

  • Synthesis of cholesterol-based cationic lipids with diverse heterocyclic head groups and linkers.
  • Assessment of cellular nucleic acid uptake using fluorescence microscopy and flow cytometry.
  • Structural analysis of lipid/nucleic acid complexes via light scattering and atomic force microscopy.

Main Results:

  • Lipids demonstrated efficient intracellular accumulation of nucleic acids.
  • Correlation found between lipid complex size (<100 nm) and transfection activity.
  • Cholesterol-based lipids with pyridine or methyl imidazole head groups and ester/carbamate linkers showed high efficacy and low toxicity.

Conclusions:

  • Biodegradable cholesterol-based lipids are effective gene delivery agents.
  • Lipid complex size is a critical factor for efficient gene transfection.
  • Specific lipid structures, particularly those with pyridine/methyl imidazole head groups, represent promising candidates for clinical gene therapy applications.