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https://link.springer.com/protocol/10.1007/978-1-61737-982-6_15
Gene delivery to the fetal muscles is a potential strategy for the early treatment of muscular dystrophies. In utero muscle gene therapy can also be used to treat other genetic disorders such as... Fetal Muscle Gene Therapy/Gene Delivery in Large Animals SpringerLinkAuthor: Khalil N. Abi-Nader, Anna L. David
https://www.researchgate.net/publication/49717168_Fetal_Muscle_Gene_TherapyGene_Delivery_in_Large_Animals
Fetal Muscle Gene Therapy/Gene Delivery in Large Animals. Gene delivery to the fetal muscles is a potential strategy for the early treatment of muscular dystrophies. In utero muscle gene therapy can also be used to treat other genetic disorders such as hemophilia, where the missing clotting proteins may be secreted from the treated muscle.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2765825/
Animal Models of DMD. There are two naturally occurring animal models for DMD, X-linked mdx mice and X-linked muscular dystrophy dogs (cxmd 1).The mdx mouse carries a single-point mutation in exon 23 of the dystrophin gene that results in a premature stop codon (Sicinski et al. 1989).Despite the absence of dystrophin expression in muscle, young mdx mice display a very mild phenotype, apart ...Author: Zejing Wang, Jeffrey S. Chamberlain, Stephen J. Tapscott, Rainer Storb
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4560173/
Jun 10, 2015 · This finding is of consequence for diseases such as cystic fibrosis, where therapeutic expression is required in the epithelia of visceral tissues, such as the intestines and in the airways and it may also be of significance in developing gene therapy strategies for skin conditions where gene delivery to a large surface area has been technically challenging.Author: Citra N. Mattar, Andrew M. S. Wong, Klemens Hoefer, Maria E. Alonso-Ferrero, Suzanne M. K. Buckley, ...
https://www.sciencedirect.com/science/article/pii/S0169409X09001458
Most experiments using fetal gene therapy have thus far been performed in animals (i.e. mice, sheep, dogs and non-human primates [66]). Mice offer the great advantage of ease of maintenance and breeding and the availability of a range of models for human genetic disease.Author: Anna M. Wagner, Andreina Schoeberlein, Daniel Surbek
https://www.nature.com/articles/3302375
Sep 29, 2004 · Transgene delivery and expression in the fetal or neonatal period is a useful tool for studying human models. One day, it may even be used therapeutically alongside adult gene therapy as a means to prevent or ameliorate monogenetic diseases.Author: S. N. Waddington, N. L. Kennea, S. M. K. Buckley, L. G. Gregory, M. Themis, C. Coutelle
https://www.sciencedirect.com/science/article/pii/S1521693407001411
Fetal gene therapy has been proposed to be appropriate for life-threatening disorders, in which prenatal gene delivery maintains a clear advantage over cell transplantation or postnatal gene therapy and for which there are currently no satisfactory treatments available. 13 Some of the diseases that may be suitable for fetal treatment are listed in Table 1. Preclinical studies are encouraging.Author: Anna L. David, Donald Peebles
https://www.intechopen.com/books/gene-therapy-applications/fetal-gene-therapy
Dog models of hemophilia with congenital deficiency [31, 32] and mouse models obtained by gene targeting and knockout technology are available to study FVIII function and gene therapy approaches for treating hemophilia A. Therapeutic benefit has been obtained in numerous studies using a variety of vector systems in the murine model [34-39], but phenotypic correction of hemophilia A in the dog has …Author: Christopher Porada, Graça Almeida-Porada
https://www.sciencedirect.com/science/article/pii/S0002937805009804
Duchenne muscular dystrophy (DMD) is a lethal degenerative muscular disease. Fetal gene therapy may correct the primary genetic defect. Our aim was to achieve expression of a reporter gene in the respiratory muscles of early gestation fetal sheep.Author: Boaz Weisz, Anna L. David, Lisa G. Gregory, Dany Perocheau, Ali Ruthe, Simon N. Waddington, Mike The...
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