Engineering human chromosomes for gene therapy studies

2001 ◽  
Vol 7 (6) ◽  
pp. 235-237 ◽  
Author(s):  
K.H.Andy Choo
Biomedicines ◽  
2021 ◽  
Vol 9 (11) ◽  
pp. 1645
Author(s):  
Bart De Geest ◽  
Mudit Mishra

Under physiological circumstances, there is an exquisite balance between reactive oxygen species (ROS) production and ROS degradation, resulting in low steady-state ROS levels. ROS participate in normal cellular function and in cellular homeostasis. Oxidative stress is the state of a transient or a persistent increase of steady-state ROS levels leading to disturbed signaling pathways and oxidative modification of cellular constituents. It is a key pathophysiological player in pathological hypertrophy, pathological remodeling, and the development and progression of heart failure. The heart is the metabolically most active organ and is characterized by the highest content of mitochondria of any tissue. Mitochondria are the main source of ROS in the myocardium. The causal role of oxidative stress in heart failure is highlighted by gene transfer studies of three primary antioxidant enzymes, thioredoxin, and heme oxygenase-1, and is further supported by gene therapy studies directed at correcting oxidative stress linked to metabolic risk factors. Moreover, gene transfer studies have demonstrated that redox-sensitive microRNAs constitute potential therapeutic targets for the treatment of heart failure. In conclusion, gene therapy studies have provided strong corroborative evidence for a key role of oxidative stress in pathological remodeling and in the development of heart failure.


2008 ◽  
Vol 40 (2) ◽  
pp. 262
Author(s):  
Annette Deichman ◽  
Manfred Schmidt ◽  
Salima Hacein-Bey Abina ◽  
Marina Cavazzana-Calvo ◽  
Kerstin Schwarzwaelder ◽  
...  

1995 ◽  
Vol &NA; (976) ◽  
pp. 3-4
Author(s):  
Lawrence M Prescott

2008 ◽  
Vol 40 (2) ◽  
pp. 258
Author(s):  
A. Jacome ◽  
P. Río ◽  
S. Navarro ◽  
A. González ◽  
J.A. Casado ◽  
...  

The Lancet ◽  
1997 ◽  
Vol 350 (9089) ◽  
pp. 1451
Author(s):  
Michael McCarthy
Keyword(s):  

2010 ◽  
Vol 125 (6) ◽  
pp. 533-537 ◽  
Author(s):  
Akira Ishiwata ◽  
Jun Mimuro ◽  
Hiroaki Mizukami ◽  
Yuji Kashiwakura ◽  
Atsushi Yasumoto ◽  
...  

Blood ◽  
2021 ◽  
Author(s):  
Frank W.G. Leebeek ◽  
Wolfgang Miesbach

In the past decade enormous progress has been made in the development of gene therapy for hemophilia A and B. After the first encouraging results of intravenously administered AAV-based liver-directed gene therapy in patients with severe hemophilia B were reported in 2011, many gene therapy studies have been initiated. Most of these studies, using AAV vectors with various gene constructs, showed sufficient FVIII and FIX expression in patients to significantly reduce the number of bleeds and the need for prophylaxis in the fast majority of the severe hemophilia patients. This resulted in great clinical benefit for nearly all patients. In this review we will summarize the most recent findings of reported and ongoing gene therapy trials. We will highlight the successful outcome of trials with focus on the results of recently reported phase 1 trials and preliminary results of phase 2b/3 trials for hemophilia A and B. These new reports also reveal the impact of side effects and drawbacks associated with gene therapy. We will therefore also discuss the limitations and remaining issues of the current gene therapy approaches. These issues have to be resolved before gene therapy will be widely available for the hemophilia patient population.


2021 ◽  
Vol 21 ◽  
Author(s):  
Firoozeh Alavian ◽  
Sorayya Ghasemi

Background: Glioblastoma multiforme (GBM) is the most common and fatal type of glioma. Nanoparticles (NPs) are using new approaches for the delivery of gene therapy in the treatment of GBM. Introduction: This article was designed to review the efficacy of NPs as the targeted carriers in the gene therapy aimed at apoptosis in GBM. Method: The appropriate keywords such as nanoparticle, glioblastoma, gene therapy, apoptosis, and the related words were used to search from PubMed, ISI Web of Science, and Scopus for relevant publications up to September 4, 2020, with no language restrictions. The present systematic review was performed based on PRISMA protocol and reviewed the articles evaluating the effects of nanoparticles, carriers of various gene therapies essentials, on GBM cells apoptosis in vitro and in vivo. The selected articles were considered using specific scores on the quality of the articles. Data extraction and quality valuation were performed by two reviewers. Result: Of 101 articles retrieved, forty-two met the inclusion criteria and were, therefore, subjected to the final deduction. The most widely used NP in GBM gene therapy studies is polyamidoamine (PAMAM). The most common gene therapy approach for apoptosis in GBM is using siRNAs. Conclusions: In conclusion, these studies validated that NPs could be a practical choices to enhance the efficiency and specific delivery in gene therapies for GBM cell apoptosis. However, the choice of NP type and gene therapy mechanism affect the GBM cell apoptotic efficiency.


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