[HTML][HTML] Adeno-associated viruses (AAV) and host immunity–A race between the hare and the hedgehog

K Rapti, D Grimm - Frontiers in immunology, 2021 - frontiersin.org
Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

[HTML][HTML] Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models

K Rapti, V Louis-Jeune, E Kohlbrenner, K Ishikawa… - Molecular Therapy, 2012 - cell.com
Adeno-associated virus (AAV)-based vectors are promising gene delivery vehicles for human
gene transfer. One significant obstacle to AAV-based gene therapy is the high prevalence …

SUMO-1 Gene Transfer Improves Cardiac Function in a Large-Animal Model of Heart Failure

…, A Lee, K Ishikawa, J Aguero, K Rapti… - Science translational …, 2013 - science.org
Recently, the impact of small ubiquitin-related modifier 1 (SUMO-1) on the regulation and
preservation of sarcoplasmic reticulum calcium adenosine triphosphatase (SERCA2a) function …

Cell-specific CRISPR–Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins

…, S Aschenbrenner, S Grosse, K Rapti… - Nucleic acids …, 2019 - academic.oup.com
The rapid development of CRISPR–Cas technologies brought a personalized and targeted
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …

[HTML][HTML] Pulmonary targeting of adeno-associated viral vectors by next-generation sequencing-guided screening of random capsid displayed peptide libraries

…, L Lunding, E Spies, A Hunger, M Alawi, K Rapti… - Molecular therapy, 2016 - cell.com
Vectors mediating strong, durable, and tissue-specific transgene expression are mandatory
for safe and effective gene therapy. In settings requiring systemic vector administration, the …

[HTML][HTML] Cardiac I-1c overexpression with reengineered AAV improves cardiac function in swine ischemic heart failure

K Ishikawa, KM Fish, L Tilemann, K Rapti, J Aguero… - Molecular therapy, 2014 - cell.com
Cardiac gene therapy has emerged as a promising option to treat advanced heart failure (HF).
Advances in molecular biology and gene targeting approaches are offering further novel …

Targeted gene therapy for the treatment of heart failure

K Rapti, AH Chaanine, RJ Hajjar - Canadian Journal of Cardiology, 2011 - Elsevier
Chronic heart failure is one of the leading causes of morbidity and mortality in Western countries
and is a major financial burden to the health care system. Pharmacologic treatment and …

[HTML][HTML] Stimulating myocardial regeneration with periostin Peptide in large mammals improves function post-myocardial infarction but increases myocardial fibrosis

D Ladage, E Yaniz-Galende, K Rapti, K Ishikawa… - PloS one, 2013 - journals.plos.org
Aims Mammalian myocardium has a finite but limited capacity to regenerate. Experimentally
stimulating proliferation of cardiomyocytes with extracellular regeneration factors like …

[HTML][HTML] Effectiveness of gene delivery systems for pluripotent and differentiated cells

K Rapti, F Stillitano, I Karakikes… - … therapy Methods & …, 2015 - cell.com
Human embryonic stem cells (hESC) and induced pluripotent stem cells (hiPSC) assert a
great future for the cardiovascular diseases, both to study them and to explore therapies. …

[HTML][HTML] Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy

…, L Schraft, A Chan, L Küchenhoff, K Rapti… - Nature …, 2023 - nature.com
Dilated cardiomyopathy is the second most common cause for heart failure with no cure
except a high-risk heart transplantation. Approximately 30% of patients harbor heritable …