Related Experiment Video
Updated: Jul 6, 2026
10:37
Decellularization-Based Quantification of Skeletal Muscle Fatty Infiltration
Published on: June 9, 2023
Muscle and fat mass modulation in different clinical models.
Ruxandra Draghia-Akli1, Amir S Khan
1VGX Pharmaceuticals, Immune Therapeutics Division, The Woodlands, TX, USA.
Methods in Molecular Biology (Clifton, N.J.)
|March 29, 2008
Summary
Gene therapy using plasmid delivery and electroporation shows promise for treating muscle and fat conditions. This cost-effective approach, delivered via injection, could offer new therapeutic options for various diseases.
Area of Science:
- Biotechnology
- Molecular Biology
- Gene Therapy
Background:
- Gene therapy using plasmid delivery and electroporation is emerging as a viable therapeutic strategy.
- Muscle-targeted gene transfer offers a cost-effective, pharmaceutical-grade therapy via intramuscular injection.
Purpose of the Study:
- To review recent innovations in plasmid-mediated gene therapy for muscle and fat modulation.
- To explore the potential of this approach for treating cachexia, autoimmune diseases, appetite disorders, and diabetes.
Main Methods:
- Plasmid DNA delivery.
- Electroporation techniques, including in situ electroporation.
- Intramuscular injection for targeted gene transfer.
Main Results:
- Gene therapy with plasmid delivery and electroporation can yield significant clinical benefits.
- In situ electroporation enables sustained systemic protein delivery within therapeutic ranges.
Conclusions:
- Plasmid-mediated gene therapy holds potential for treating a range of conditions affecting muscle and fat.
- Advancements in gene therapy techniques are paving the way for human clinical trials and viable therapeutic alternatives.
Related Concept Videos
01:26Pharmacokinetic Models: Comparison and Selection Criterion
Physiological and compartmental models are valuable tools used in studying biological systems. These models rely on differential equations to maintain mass balance within the system, ensuring an accurate representation of the dynamic processes at play.
Physiological models take a detailed approach by considering specific molecular processes. They can predict drug distribution, metabolism, and elimination changes, providing a comprehensive understanding of how drugs interact with the body.
Physiological models take a detailed approach by considering specific molecular processes. They can predict drug distribution, metabolism, and elimination changes, providing a comprehensive understanding of how drugs interact with the body.
01:25Pharmacokinetics in Obese Patients: Drug Absorption and Distribution
Obesity significantly alters the pharmacokinetic processes of drug absorption and distribution, presenting unique challenges in medical treatment. The increased fat tissue and decreased lean muscle in obese individuals can significantly affect how drugs are absorbed into the body and distributed across different tissues. This alteration can lead to variances in the effectiveness and safety of medications, necessitating adjustments in dosing or drug selection for obese patients.One notable...
01:09Clearance Models: Physiological Models
Drug clearance is a critical pharmacokinetic process involving the irreversible removal of drugs from the body through various organs over a specified time period. Physiological models are indispensable in determining organ-specific clearance, defined by the proportion of the drug eliminated per unit of time from the organ's blood volume.
The organ's clearance rate depends on the blood flow to the organ and the extraction ratio (E). The extraction ratio describes the organ's proficiency in drug...
The organ's clearance rate depends on the blood flow to the organ and the extraction ratio (E). The extraction ratio describes the organ's proficiency in drug...
01:15Model Approaches for Pharmacokinetic Data: Physiological Models
Physiological models in pharmacokinetics are instrumental in understanding the distribution and elimination of drugs within the body. These models describe the drug concentration within target organs, influenced by factors such as drug uptake, tissue volume, and blood flow. Drug uptake is governed by the partition coefficient, which signifies the drug concentration ratio in tissue to that in the blood. The blood flow rate to a specific tissue is expressed as Qt, and the rate of change in tissue...
01:20Pharmacokinetic Models: Overview
Pharmacokinetic models utilize mathematical analysis to achieve a detailed quantitative understanding of a drug's life cycle within the body. They are instrumental in simulating a drug's pharmacokinetic parameters, predicting drug concentrations over time, optimizing dosage regimens, linking concentrations with pharmacologic activity, and estimating potential toxicity.
There are three primary types of models: empirical, compartment, and physiological. Empirical models, with minimal assumptions,...
There are three primary types of models: empirical, compartment, and physiological. Empirical models, with minimal assumptions,...
01:13Physiological Pharmacokinetic Models: Assumption with Protein Binding
Physiological models with protein binding in pharmacokinetics offer a sophisticated approach to understanding drug disposition. These models consider drug-protein interactions, enabling them to effectively predict drug concentrations in different organs and tissues. This precision aids in accurate drug dosing, providing a significant advantage over conventional models. A key process within these models is equilibration, which ensures that drug concentrations achieve a steady state within the...