Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy.

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Research Abstract Details 

Research Abstract Table of Contents

Jump to the:

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Abstract Text:

The number of diseases found to be associated with defects of the mitochondrial genome has grown significantly over the last decade. Despite major advances in understanding mtDNA defects at the genetic and biochemical level, there is no satisfactory treatment for the vast majority of patients available. This is largely due to the fact that almost all mitochondrial DNA defects involve the final common pathway of oxidative metabolism making it impossible to bypass the defect by giving alternative metabolic carriers of energy. These seemingly objective limitations of conventional biochemical treatment for patients with detects of mtDNA warrant the exploration of gene therapeutic approaches. However, mitochondrial gene therapy still appears only theoretical and speculative. Any possibility for gene replacement is dependent on the use of a yet unavailable mitochondria-specific transfection vector. Based upon an analysis of the self-assembly behavior of dequalinium, a cationic single-chain bolaamphiphile which is known to selectively accumulate in mitochondria, we have developed a whole new strategy for mitochondria-specific DNA delivery. We have succeeded in preparing vesicles made of dequalinium, which we termed DQAsomes (U.S. Patent 6,090,619). We have shown that DQAsomes efficiently bind and protect DNA and we could demonstrate that DQAsome/DNA complexes selectively release DNA at cardiolipin-rich liposomes mimicking both, the inner and the outer mitochondrial membrane. Based on the intrinsic property of dequalinium to preferentially accumulate in mitochondria in response to the electrochemical gradient at the mitochondrial membrane and based on the selective DNA release at mitochondria-like membranes we propose DQAsomes as the first mitochondria-specific vector to deliver DNA to mitochondria in living cells.

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Publishing Authors By Initials

For similar proteins research abstracts see: proteins research

PUBMED ID PMID:

MEDLINE DATE:

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Journal Published:

PUBLICATION TYPE: Review

Journal: Current pharmaceutical biotechnology

VOLUME: 1

Page Numbers: 325-46

Journal Abbreviation: Curr Pharm Biotechnol

ISSN: 1389-2010

DAY: 12

MONTH: Dec

YEAR: 2000

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Information

Number of References: 97

LANGUAGE: eng

NlmUniqueID: 100960530

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Keywords Mesh Terms:

KEYWORDS: Proteins

MESH TERMS: metabolism

Chemical & Substance for Abstract: Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy. Information

Substance Name: Proteins

Registry Number: 0

Grant and Affiliation Information for Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy.

AFFILIATION: Northeastern University, Bouve College of Health Sciences School of Pharmacy, Department of Pharmaceutical Sciences, Boston, MA 02115, USA. vweissig@hotmail.com

Country: Netherlands

AGENCY:

GRANT:

ACRONYM:

MEDLINETA: Curr Pharm Biotechnol

REFSOURCE:

DATABASENAME:

ACCESSION NUMBER:

Number Hits: 0

Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy Related Publications

• "SMART" drug delivery systems: double-targeted pH-responsive pharmaceutical nanocarriers.
• "Smart" Drug Carriers: PEGylated TATp-Modified pH-Sensitive Liposomes.
• Antibody-modified liposomes for cancer chemotherapy.
• Antinucleosome antibody-modified liposomes and lipid-core micelles for tumor-targeted delivery of therapeutic and diagnostic agents.
• Bioengineering and Imaging Research Opportunities Workshop V: A summary on Imaging and Characterizing Structure and Function in Native and Engineered Tissues.
• Bioengineering and Imaging Research Opportunities Workshop V: a white paper on imaging and characterizing structure and function in native and engineered tissues.
• Bioengineering and imaging research opportunities workshop V: summary of findings on imaging and characterizing structure and function in native and engineered tissues.
• Bioengineering and imaging research opportunities workshop v: a summary.
• Design, synthesis, and characterization of pH-sensitive PEG-PE conjugates for stimuli-sensitive pharmaceutical nanocarriers: the effect of substitutes at the hydrazone linkage on the ph stability of PEG-PE conjugates.
• Enhanced accumulation of long-circulating liposomes modified with the nucleosome-specific monoclonal antibody 2C5 in various tumours in mice: gamma-imaging studies.
• Enhanced cytotoxicity of monoclonal anticancer antibody 2C5-modified doxorubicin-loaded PEGylated liposomes against various tumor cell lines.
• Enhanced tumor MR imaging with gadolinium-loaded polychelating polymer-containing tumor-targeted liposomes.
• Enhanced tumor visualization by gamma-scintigraphy with 111In-labeled polychelating-polymer-containing immunoliposomes.
• Mitochondriotropic cationic vesicles: a strategy towards mitochondrial gene therapy.
• Mixed PEG-PE/vitamin E tumor-targeted immunomicelles as carriers for poorly soluble anti-cancer drugs: Improved drug solubilization and enhanced in vitro cytotoxicity.
• Monoclonal antibody 2C5-modified doxorubicin-loaded liposomes with significantly enhanced therapeutic activity against intracranial human brain U-87 MG tumor xenografts in nude mice.
• Nanocarriers.
• PEGylated dextran as long-circulating pharmaceutical carrier.
• Photodynamic therapy of experimental B-16 melanoma in mice with tumor-targeted 5,10,15,20-tetraphenylporphin-loaded PEG-PE micelles.
• Polyethylene glycol-phosphatidylethanolamine conjugate (PEG-PE)-based mixed micelles: some properties, loading with paclitaxel, and modulation of P-glycoprotein-mediated efflux.
• Polymeric contrast agents for medical imaging.
• Preface. Fifth International Nanomedicine and Drug Delivery Symposium (NanoDDS'07).
• Protective effect of coenzyme Q10-loaded liposomes on the myocardium in rabbits with an acute experimental myocardial infarction.
• Stable nanocolloids of poorly soluble drugs with high drug content prepared using the combination of sonication and layer-by-layer technology.
• Surface Modification of Pharmaceutical Nanocarriers with Ascorbate Residues Improves their Tumor-Cell Association and Killing and the Cytotoxic Action of Encapsulated Paclitaxel In Vitro.
• TAT peptide-modified liposomes provide enhanced gene delivery to intracranial human brain tumor xenografts in nude mice.
• Tat peptide-mediated intracellular delivery of pharmaceutical nanocarriers.
• Tatp-mediated intracellular delivery of pharmaceutical nanocarriers.
• The architecture of ligand attachment to nanocarriers controls their specific interaction with target cells.
• Tumor-specific antibody-mediated targeted delivery of Doxil((R)) reduces the manifestation of auricular erythema side effect in mice.