scholarly journals Gene Therapy in Large Animal Models of Human Cardiovascular Genetic Disease

ILAR Journal ◽  
2009 ◽  
Vol 50 (2) ◽  
pp. 199-205 ◽  
Author(s):  
M. M. Sleeper ◽  
L. T. Bish ◽  
H. L. Sweeney
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Genetic Disease ◽  
Large Animal ◽  
Large Animal Models

scholarly journals Intratracheal aerosolization of viral vectors to newborn pig airways

BioTechniques ◽  
2020 ◽  
Vol 68 (5) ◽  
pp. 235-239
Author(s):  
Ashley L Cooney ◽  
Patrick L Sinn
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Endotracheal Tube ◽  
Viral Vectors ◽  
Small Animal ◽  
Large Animal ◽  
Large Animal Models ◽  
Airway Diseases ◽  
Efficient Delivery ◽  
Large Animals

Gene therapy for airway diseases requires efficient delivery of nucleic acids to the airways. In small animal models, gene delivery reagents are commonly delivered as a bolus dose. However, large animal models are often more relevant for the transition from preclinical studies to human trials. Aerosolizing viral vectors to the lungs of large animals can maximize anatomical distribution. Here, we describe a technique for aerosolization of viral vectors to the airways of newborn pigs. Briefly, a pig is anesthetized and intubated with an endotracheal tube, and a microsprayer is passed through the endotracheal tube. A fine mist is then sprayed into the distal trachea. Widespread and uniform distribution of transgene expression is critical for developing successful lung gene therapy treatments.

scholarly journals Large animal models and gene therapy

2005 ◽  
Vol 14 (3) ◽  
pp. 266-272 ◽  
Author(s):  
Margret Casal ◽  
Mark Haskins
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Large Animal ◽  
Large Animal Models

scholarly journals Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials

2011 ◽  
Vol 2 ◽  
Author(s):  
Zejing Wang ◽  
Stephen J. Tapscott ◽  
Jeffrey S. Chamberlain ◽  
Rainer Storb
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Muscular Dystrophies ◽  
Large Animal ◽  
Human Trials ◽  
Large Animal Models

scholarly journals Large Animal Models of Genetic Disease: Pertinent IACUC Issues

ILAR Journal ◽  
2009 ◽  
Vol 50 (2) ◽  
pp. 225-228 ◽  
Author(s):  
N. M. Ellinwood ◽  
C. M. Clay
Keyword(s):  
Animal Models ◽  
Genetic Disease ◽  
Large Animal ◽  
Large Animal Models

scholarly journals Potential Large Animal Models for Gene Therapy of Human Genetic Diseases of Immune and Blood Cell Systems

ILAR Journal ◽  
2009 ◽  
Vol 50 (2) ◽  
pp. 168-186 ◽  
Author(s):  
T. R. Bauer ◽  
R. L. Adler ◽  
D. D. Hickstein
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Blood Cell ◽  
Genetic Diseases ◽  
Large Animal ◽  
Cell Systems ◽  
Large Animal Models

Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Use of Large Animal Models with Relevance to Human Application

2012 ◽  
pp. 291-328 ◽  
Author(s):  
Vedanta Mehta ◽  
Khalil N. Abi-Nader ◽  
David Carr ◽  
Jacqueline Wallace ◽  
Charles Coutelle ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Adverse Effects ◽  
Large Animal ◽  
Large Animal Models

scholarly journals Systematic Review of the Preclinical Technology Readiness of Orthopedic Gene Therapy and Outlook for Clinical Translation

2021 ◽  
Vol 9 ◽  
Author(s):  
Piers Wilkinson ◽  
Ilya Y. Bozo ◽  
Thomas Braxton ◽  
Peter Just ◽  
Elena Jones ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Animal Models ◽  
Bone Repair ◽  
Safety Data ◽  
Clinical Translation ◽  
Model Systems ◽  
Large Animal ◽  
Repair Gene ◽  
Public Health Burden ◽  
Large Animal Models

Bone defects and improper healing of fractures are an increasing public health burden, and there is an unmet clinical need in their successful repair. Gene therapy has been proposed as a possible approach to improve or augment bone healing with the potential to provide true functional regeneration. While large numbers of studies have been performed in vitro or in vivo in small animal models that support the use of gene therapy for bone repair, these systems do not recapitulate several key features of a critical or complex fracture environment. Larger animal models are therefore a key step on the path to clinical translation of the technology. Herein, the current state of orthopedic gene therapy research in preclinical large animal models was investigated based on performed large animal studies. A summary and an outlook regarding current clinical studies in this sector are provided. It was found that the results found in the current research literature were generally positive but highly methodologically inconsistent, rendering a comparison difficult. Additionally, factors vital for translation have not been thoroughly addressed in these model systems, and the risk of bias was high in all reviewed publications. These limitations directly impact clinical translation of gene therapeutic approaches due to lack of comparability, inability to demonstrate non-inferiority or equivalence compared with current clinical standards, and lack of safety data. This review therefore aims to provide a current overview of ongoing preclinical and clinical work, potential bottlenecks in preclinical studies and for translation, and recommendations to overcome these to enable future deployment of this promising technology to the clinical setting.

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