Duchenne Muscular Dystrophy (DMD) is a devastating progressive muscle wasting disorder caused by mutations on the DMD gene that result in the complete absence of its protein product, dystrophin. Studies involving Adeno-associated viral (AAV) vector-mediated gene transfer approaches highlight the challenges the immune system can impose when it encounters “non-self” dystrophin epitopes. As an alternative, our lab relies on utrophin, a dystrophin paralog that retains most of dystrophin’s structural and binding elements. Importantly, normal thymic expression in DMD subjects should protect utrophin by central immunologic tolerance. Here I describe how an AAV vector containing a codon optimized truncated utrophin transgene (AAV-μUtrophin) is a hi...
Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a degenerative, le...
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a s...
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It i...
Duchenne Muscular Dystrophy (DMD) is a devastating progressive muscle wasting disorder caused by mut...
Duchenne muscular dystrophy (DMD) is a fatal disease caused by defects in the gene encoding dystroph...
Utrophin is a close homolog of dystrophin, the protein whose mutations cause Duchenne muscular dystr...
Recombinant adeno-associated virus (rAAV) based vectors have emerged as widely used gene transfer ve...
Recombinant adeno-associated virus (rAAV) based vectors have emerged as widely used gene transfer ve...
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a s...
Helper-dependent adenovirus vector (AdV)\u2013mediated full-length dystrophin expression leads to si...
Thesis (Ph.D.)--University of Washington, 2014Duchenne muscular dystrophy (DMD) is a recessive muscl...
Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a degenerative, le...
Dystrophin gene transfer using gutted or helper-dependent adenoviruses (HDAd), which have most of th...
First-generation adenovirus (Ad) vectors are unsuitable for gene replacement therapy, due to immune ...
Helper-dependent adenovirus vector (AdV)-mediated full-length dystrophin expression leads to signifi...
Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a degenerative, le...
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a s...
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It i...
Duchenne Muscular Dystrophy (DMD) is a devastating progressive muscle wasting disorder caused by mut...
Duchenne muscular dystrophy (DMD) is a fatal disease caused by defects in the gene encoding dystroph...
Utrophin is a close homolog of dystrophin, the protein whose mutations cause Duchenne muscular dystr...
Recombinant adeno-associated virus (rAAV) based vectors have emerged as widely used gene transfer ve...
Recombinant adeno-associated virus (rAAV) based vectors have emerged as widely used gene transfer ve...
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a s...
Helper-dependent adenovirus vector (AdV)\u2013mediated full-length dystrophin expression leads to si...
Thesis (Ph.D.)--University of Washington, 2014Duchenne muscular dystrophy (DMD) is a recessive muscl...
Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a degenerative, le...
Dystrophin gene transfer using gutted or helper-dependent adenoviruses (HDAd), which have most of th...
First-generation adenovirus (Ad) vectors are unsuitable for gene replacement therapy, due to immune ...
Helper-dependent adenovirus vector (AdV)-mediated full-length dystrophin expression leads to signifi...
Duchenne muscular dystrophy (DMD), the most common form of muscular dystrophy, is a degenerative, le...
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disorder caused by the lack of a s...
Duchenne muscular dystrophy (DMD) is a serious, rare genetic disease, affecting primarily boys. It i...