Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

Monahan, Paul E.; Samulski, R. Jude; Tazelaar, John; Xiao, Xiao; Nichols, Timothy C.; Bellinger, Dwight A.; Read, Marjorie S.; Walsh, Christopher E.

doi:10.1038/sj.gt.3300548

Cited by 189 publications

(118 citation statements)

References 32 publications

(40 reference statements)

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“…[18][19][20][21] In treated muscle of immunocompetent mice, this expression was shown to persist for 1.5 years. 19 Potentially therapeutic proteins that have been shown to be delivered and expressed by AAV vectors include erythropoietin, 20 cystic fibrosis transmembrane conductance regulator, 22 blood coagulation factor IX, 23,24 and GUS. 21 In none of these cases, however, were experiments designed to show that the AAV-mediated gene therapy actually affected the long-term course of a disease.…”

Section: Introductionmentioning

confidence: 99%

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

Watson¹,

Sayles²,

Chen³

et al. 1998

Gene Ther

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Mucopolysaccharidosis type VII (MPS VII) is a lysosomalproduced low GUS activity in several tissues. This latter storage disease caused by a genetic deficiency of ␤-glucutreatment of MPS VII mice reduced glycosaminoglycan ronidase (GUS). We used a recombinant adeno-associalevels in the liver to normal and reduced storage granules ted virus vector (AAV-GUS) to deliver GUS cDNA to MPS dramatically. We show that a single administration of VII mice. The route of vector administration had a dramatic AAV-GUS can provide sustained expression of GUS in a effect on the extent and distribution of GUS activity. Intravariety of cell types and is sufficient to reverse the disease muscular injection of AAV-GUS resulted in high, localized phenotype at least in the liver. production of GUS, while intravenous administration

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Section: Introductionmentioning

confidence: 99%

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

Watson¹,

Sayles²,

Chen³

et al. 1998

Gene Ther

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“…31,32 It has been shown in animal models that rAAV is an attractive in vivo gene delivery vehicle. [33][34][35][36][37][38][39][40] For example, a single injection of rAAV into the muscle of mice resulted in sustained transgene expression for over 1.5 years and did not elicit a cytotoxic T cell response against the virus or the transgene. 33 rAAV has previously been shown to transduce several epitheloid cell lines efficiently.…”

mentioning

confidence: 99%

Efficient gene transfer into human keratinocytes with recombinant adeno-associated virus vectors

Braun‐Falco

Doenecke

Smola³

et al. 1999

Gene Ther

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Gene transfer into the skin is a promising approach to treat duced within 48 h. This effect was independent of individinherited or acquired dermatological diseases and sysual skin donors and different body areas serving as the temic monogenic deficiencies. For this purpose, the source for keratinocyte isolation. rAAV had no significant efficient and sustained gene delivery into keratinocytes is influence on cell viability, but induced a growth arrest in of critical importance. Recombinant adeno-associated transduced keratinocytes. This growth arrest was overvirus (rAAV) vectors hold the potential to achieve a longcome by replating cells in fresh media. rAAV/GFP-transterm gene transfer into various human organs. In order to duced keratinocytes could be passaged several times, evaluate this potential for skin gene therapy, human keraexpressed GFP for up to 50 days, and passed the transtinocytes were transduced in vitro with rAAV vectors encogene to their daughter cells, suggesting that keratinocyte ding the reporter genes ␤-galactosidase (rAAV/LacZ) or precursor cells were also transduced. Taken together, the green fluorescent protein (rAAV/GFP). Using rAAV/LacZ at results suggest that rAAV is a promising gene transfer a multiplicity of infection (MOI) of five transducing particles vehicle for skin gene therapy. per cell, up to 70% of human keratinocytes were trans-

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“…AAV has also been shown to use heparan sulfate as a co-receptor to gain entry to cells, 49,50 and is capable of infecting mature myofibers quite efficiently. [51][52][53][54] There are several possibilities to account for this difference. The relatively small size of AAV compared with HSV (20 nm versus 200 nm) may allow it to penetrate the basal lamina better to engage its cognate receptors.…”

Section: Discussionmentioning

confidence: 99%

Efficient infection of mature skeletal muscle with herpes simplex virus vectors by using dextran sulfate as a co-receptor

1999

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The use of herpes simplex virus (HSV) vectors for gene chondroitin ABC lyase, HSV infection was restored, which delivery to skeletal muscle is hampered by a maturationsuggests that virus secondary receptors were present but dependent loss of muscle fiber infectivity. Previous studies not readily accessible to the virus in the intact myofiber. of HSV type 1 (HSV-1) infection in the rodent show that Surprisingly, we also found that HSV-1 infectivity could be the loss of infectivity may be due, at least in part, to the restored in vitro and in vivo by exposing myofibers to low development of the basal lamina throughout the course of concentrations of the glycosaminoglycan analog dextran maturation, which may block the initial events in HSV infecsulfate, which appears to act as a surrogate receptor to tion. To initiate infection, HSV normally attaches to cell surstabilize the virus at the myofiber surface such that HSV face heparan sulfate, which stabilizes the virus such that can engage additional receptors. This demonstration that it can interact with secondary protein receptors required the basal lamina is not an absolute block to HSV-1 infecfor entry into host cells. In this study, we demonstrate that tion is remarkable because it allows for the nondestructive heparan sulfate biosynthesis is downregulated during skeltargeting of HSV-1 to mature myofibers and greatly etal muscle maturation. When myofibers were treated with expands the usefulness of HSV as a gene therapy vector a variety of enzymes, including collagenase type IV or for the treatment of inherited and acquired diseases.

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Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

Cited by 189 publications

References 32 publications

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

Efficient gene transfer into human keratinocytes with recombinant adeno-associated virus vectors

Efficient infection of mature skeletal muscle with herpes simplex virus vectors by using dextran sulfate as a co-receptor

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