|
Adult Glioblastoma
|
0.010 |
Biomarker
|
disease |
BEFREE |
The latter is of particular interest because the AAV integration site (AAVS1) is located on the long arm of chromosome 19 and 30-40% of human glioblastoma tumors are reported to have loss of heterozygosity in this region of chromosome 19q.
|
12542846 |
2002 |
|
Allergy to peanuts
|
0.010 |
Biomarker
|
phenotype |
BEFREE |
Based on the knowledge that peanut allergy is mediated by peanut-specific IgE, we hypothesized that a single administration of an adeno-associated virus (AAV) gene transfer vector encoding for anti-hIgE would protect against repeated peanut exposure in the host with peanut allergy.
|
27372563 |
2016 |
|
alpha 1-Antitrypsin Deficiency
|
0.010 |
GeneticVariation
|
disease |
BEFREE |
These results support the feasibility and safety of AAV gene therapy for AAT deficiency, and indicate that serum levels of vector-derived normal human AAT >20 μg/ml can be achieved.
|
21609134 |
2011 |
|
Alpha-Sarcoglycanopathies
|
0.010 |
AlteredExpression
|
disease |
BEFREE |
The aim of this study was to attain long-lasting alpha-sarcoglycan gene expression in limb-girdle muscular dystrophy, type 2D (LGMD2D) subjects mediated by adeno-associated virus (AAV) gene transfer under control of a muscle specific promoter (tMCK).
|
21031578 |
2010 |
|
Alzheimer's Disease
|
0.010 |
GeneticVariation
|
disease |
BEFREE |
Adeno-associated virus (AAV) has been the vector of choice in recent clinical trials of neurological disease, including Parkinson's and Alzheimer's disease, due to the safety, efficacy, and stability of AAV gene transfer to the CNS.
|
22465202 |
2013 |
|
Angioedemas, Hereditary
|
0.010 |
GeneticVariation
|
disease |
BEFREE |
As an approach to effectively treat HAE with a single treatment, we hypothesized that a one-time intravenous administration of an adeno-associated virus (AAV) gene transfer vector expressing the genetic sequence of the normal human C1 esterase inhibitor (AAVrh.10hC1EI) would provide sustained circulating C1EI levels sufficient to prevent angioedema episodes.
|
30059156 |
2019 |
|
Arrhythmogenic Right Ventricular Dysplasia
|
0.010 |
GeneticVariation
|
disease |
BEFREE |
We developed a new model of cardiac tissue-specific transgenic-like mice on the basis of AAV gene transfer to test the potential of a combination of a human PKP2 mutation and endurance training to trigger an ARVC-like phenotype.
|
25857910 |
2015 |
|
Blood Coagulation Disorders
|
0.020 |
AlteredExpression
|
group |
BEFREE |
Adeno-associated viral (AAV) gene transfer of coagulation factor IX to skeletal muscle and liver of murine and canine models of hemophilia has resulted in sustained systemic expression and, in several studies, in complete cure of the bleeding disorder.
|
17266422 |
2007 |
|
Blood Coagulation Disorders
|
0.020 |
AlteredExpression
|
group |
BEFREE |
Adeno-associated viral (AAV) gene transfer of coagulation factor VIII and IX to skeletal muscle and liver of murine and canine models of hemophilia A and B have resulted in sustained systemic expression and, in several studies, in complete cure of the bleeding disorder.
|
15975012 |
2005 |
|
Carcinoma, Ovarian Epithelial
|
0.010 |
Biomarker
|
disease |
BEFREE |
To circumvent this, here we employ adeno-associated virus (AAV) gene therapy vectors to express 3TSR alone or in combination with the CD47-binding peptide of TSP-1 and evaluate the impact on tumor development and survival in a mouse model of EOC.
|
31160686 |
2019 |
|
Cardiac Arrhythmia
|
0.010 |
GeneticVariation
|
phenotype |
BEFREE |
Multiple successful preclinical studies suggest a potential utility of AAV gene therapy for arrhythmias and biological heart pacing, as well as RNA overexpression.
|
25783685 |
2015 |
|
Cardiovascular Diseases
|
0.010 |
Biomarker
|
group |
BEFREE |
The advances made in each mode of intervention as well as the ones that are beyond the scope of AAV gene therapy or has not been approached through AAV gene therapy as of now have been provided in detail to illustrate the bigger picture of where we stand to combat cardiovascular diseases most efficiently.
|
28589503 |
2017 |
|
Childhood Glioblastoma
|
0.010 |
Biomarker
|
disease |
BEFREE |
The latter is of particular interest because the AAV integration site (AAVS1) is located on the long arm of chromosome 19 and 30-40% of human glioblastoma tumors are reported to have loss of heterozygosity in this region of chromosome 19q.
|
12542846 |
2002 |
|
Childhood Osteosarcoma
|
0.010 |
Biomarker
|
disease |
BEFREE |
To construct a stable RECQL5 overexpression osteosarcoma cell line (MG-63-RECQL5), RECQL5 gene was inserted into the human AAVS1 safe harbor by CRISPR/Cas9 system.
|
26499077 |
2015 |
|
Choroideremia
|
0.020 |
GeneticVariation
|
disease |
BEFREE |
With recent advances in adeno-associated virus (AAV) gene therapy for CHM showing gene replacement to be a promising approach, an assay to assess the biological activity of the vectors is of the uttermost importance.
|
29707603 |
2018 |
|
Choroideremia
|
0.020 |
Biomarker
|
disease |
BEFREE |
Choroideremia: molecular mechanisms and development of AAV gene therapy.
|
29932012 |
2018 |
|
Chronic granulomatous disease
|
0.010 |
Biomarker
|
group |
BEFREE |
Using a standard platform that combines iPSC generation from peripheral blood CD34(+) cells and ZFN mediated AAVS1 safe harbor minigene targeting, we demonstrate efficient generation of genetically corrected iPSCs using an identical approach for all five genetic forms of CGD.
|
25288370 |
2015 |
|
Citrullinemia Type 1
|
0.010 |
Biomarker
|
disease |
BEFREE |
A murine model of CTLN1 (fold/fold) that displays lethality within the first 21 days of life was used to determine the efficacy of adeno-associated viral (AAV) gene transfer as a potential therapy.
|
24131980 |
2013 |
|
CNS disorder
|
0.010 |
GeneticVariation
|
group |
BEFREE |
The present review summarizes the progress on AAV gene delivery models for three different CNS disorders.
|
19072910 |
2008 |
|
Congestive heart failure
|
0.030 |
Biomarker
|
disease |
BEFREE |
We summarize the significant recent progress that has been made in treating heart failure in preclinically relevant animal models with AAV gene therapy and the recent results of clinical trials with cardiac AAV gene therapy for the treatment of heart failure.
|
28169951 |
2017 |
|
Congestive heart failure
|
0.030 |
AlteredExpression
|
disease |
BEFREE |
AAV gene delivery elevated the intracellular expression of the AR185 protein in a rat model of ischemic HF, and this treatment normalized the systolic and diastolic dysfunction of the failing myocardium <i>in vivo</i> by reversing myocardial Ca<sup>2+</sup> handling.
|
30885011 |
2019 |
|
Congestive heart failure
|
0.030 |
Biomarker
|
disease |
BEFREE |
Many molecular targets selected to treat heart failure using AAV gene therapy have been chosen because of their potential to regulate and restore cardiac contractility.
|
29703647 |
2018 |
|
Corneal Neovascularization
|
0.010 |
GeneticVariation
|
disease |
BEFREE |
To develop a safe and effective therapy for corneal vascularization, adeno-associated virus (AAV) gene therapy, exploiting a natural immune tolerance mechanism induced by human leukocyte antigen G (HLA-G), was investigated.
|
29259248 |
2017 |
|
Cystic Fibrosis
|
0.060 |
Biomarker
|
disease |
BEFREE |
Areas covered: Herein, the authors focus on AAV gene therapy for CF, evaluating past experience with this approach and identifying ways forward, based on the progress that has already been made in identifying and overcoming the limitations of AAV gene therapy.
|
28657358 |
2017 |
|
Cystic Fibrosis
|
0.060 |
Biomarker
|
disease |
BEFREE |
All these studies showed that AAV gene therapy for CF is safe, but clinical benefit was not clearly demonstrated.
|
28726496 |
2017 |