18658138. METHODS OF TREATING MITOCHONDRIAL DISORDERS simplified abstract (The Regents of the University of California)
Contents
METHODS OF TREATING MITOCHONDRIAL DISORDERS
Organization Name
The Regents of the University of California
Inventor(s)
Stephanie Cherqui of La Jolla CA (US)
METHODS OF TREATING MITOCHONDRIAL DISORDERS - A simplified explanation of the abstract
This abstract first appeared for US patent application 18658138 titled 'METHODS OF TREATING MITOCHONDRIAL DISORDERS
- Simplified Explanation:**
The patent application describes methods for treating diseases related to mitochondrial dysfunction by introducing a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) ex vivo and then transplanting the modified cells into a patient.
- Key Features and Innovation:**
- Treatment of diseases associated with mitochondrial dysfunction
- Introduction of nucleic acid molecules into HSPCs ex vivo
- Transplantation of modified HSPCs into patients
- Nucleic acid molecule may contain functional human frataxin (hFXN) or a gene editing system to remove mutations
- Targeting the root cause of the disease at the cellular level
- Potential Applications:**
- Treatment of mitochondrial diseases such as Friedreich's ataxia
- Potential for personalized medicine approaches
- Research in gene therapy and stem cell transplantation
- Problems Solved:**
- Addressing mitochondrial dysfunction at the cellular level
- Providing a potential cure for diseases with genetic mutations
- Improving the efficacy of stem cell transplantation therapies
- Benefits:**
- Potential for long-term disease management
- Targeted treatment at the genetic level
- Improved quality of life for patients with mitochondrial diseases
- Commercial Applications:**
Potential commercial applications include:
- Development of gene therapy treatments for mitochondrial diseases
- Licensing of the technology for pharmaceutical companies
- Research collaborations in stem cell therapy and genetic medicine
- Questions about Mitochondrial Dysfunction:**
1. How common are diseases associated with mitochondrial dysfunction? 2. What are the challenges in developing gene therapy treatments for mitochondrial diseases?
- Frequently Updated Research:**
Stay updated on the latest advancements in gene therapy for mitochondrial diseases and stem cell transplantation research for improved treatment outcomes.
Original Abstract Submitted
Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.