Methods to monitor gene therapy with molecular imaging.

Methods

Laboratory for Gene Therapy and Molecular Imaging, Max Planck Institute for Neurological Research and Faculty of Medicine, University of Cologne, Gleuelerstrasse 50, Cologne 50931, Germany.

Published: June 2009

Recent progress in scientific and clinical research has made gene therapy a promising option for efficient and targeted treatment of several inherited and acquired disorders. One of the most critical issues for ensuring success of gene-based therapies is the development of technologies for non-invasive monitoring of the distribution and kinetics of vector-mediated gene expression. In recent years many molecular imaging techniques for safe, repeated and high-resolution in vivo imaging of gene expression have been developed and successfully used in animals and humans. In this review molecular imaging techniques for monitoring of gene therapy are described and specific use of these methods in the different steps of a gene therapy protocol from gene delivery to assessment of therapy response is illustrated. Linking molecular imaging (MI) to gene therapy will eventually help to improve the efficacy and safety of current gene therapy protocols for human application and support future individualized patient treatment.

Download full-text PDF

Source
http://dx.doi.org/10.1016/j.ymeth.2009.03.007DOI Listing

Publication Analysis

Top Keywords

gene therapy
24
molecular imaging
16
gene
9
gene expression
8
imaging techniques
8
imaging gene
8
therapy
7
imaging
5
methods monitor
4
monitor gene
4

Similar Publications

Therapeutic Potential of Carbon Dots Derived from Phytochemicals as Nanozymes Exhibiting Superoxide Dismutase Activity for Anemia.

ACS Appl Mater Interfaces

January 2025

State Key Laboratory of Natural and Biomimetic Drugs, School of Pharmaceutical Sciences, Peking University, Beijing 100191, China.

Anemia is a potentially life-threatening blood disorder caused by an insufficient erythroblast volume in the circulatory system. Self-renewal failure of erythroblast progenitors is one of the key pathological factors leading to erythroblast deficiency. However, there are currently no effective drugs that selectively target this process.

View Article and Find Full Text PDF

Ganaxolone: A Review in Epileptic Seizures Associated with Cyclin-Dependent Kinase-Like 5 Deficiency Disorder.

Paediatr Drugs

January 2025

Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.

Oral ganaxolone (ZTALMY), a synthetic analogue of the endogenous neuroactive steroid allopregnanolone, acts as a positive allosteric modulator of synaptic and extra-synaptic γ-aminobutyric acid (GABA) type A receptor function in the CNS. In the EU and the UK, it is approved for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2-17 years. In a multinational phase III study (Marigold), 17 weeks' therapy with adjunctive ganaxolone, administered orally three times daily with food, significantly reduced 28-day major motor seizure frequency from baseline versus placebo in patients aged 2-19 years with CDD-associated refractory epilepsy.

View Article and Find Full Text PDF

Targeted therapy has emerged as a promising option in cancer treatment, driven by advances in the understanding of DNA changes and the molecular basis of cancer. This article provides an overview of next-generation sequencing and types of genetic alterations, common cancer biomarkers, a review of circulating tumor DNA testing and its applications for oncology treatments, how to read a genomic testing report, examples of targeted therapy for cancer pathologic variants and tumor markers, and the implications for nursing practice in this emerging field.

View Article and Find Full Text PDF

Purpose: The purpose of this study was to analyze the retinal sensitivity under photopic, mesopic, and scotopic conditions in a cohort of patients affected with KCNV2-associated retinopathy.

Methods: Cross-sectional evaluation of molecularly confirmed individuals was conducted. Data were obtained prospectively.

View Article and Find Full Text PDF

Tamoxifen is an inhibitor of estrogen receptors and was originally developed for breast cancer therapy. Besides, tamoxifen is widely used for Cre-estrogen receptor-mediated conditional knockout in transgenic mice. However, we found that the 3-month feeding of 0.

View Article and Find Full Text PDF

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!