Highly Efficient<i>Ex Vivo</i>Gene Delivery into Human Corneal Endothelial Cells by Recombinant Adeno-Associated Virus

Lai, Li; Lin, Ken Kuo; Foulks, Gary N.; Ma, Lih; Xiao, Xiao; Chen, Ko Hua

doi:10.1080/02713680590927515

Cited by 19 publications

(22 citation statements)

References 23 publications

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“…confirmed previous studies showing that this viral vector has stable and long-term expression limited to the liver, skeletal, and cardiac muscle (McClane et al 1997, Song et al 2001, Tsai et al 2002, Ayuso et al 2004, Arruda et al 2005, Lai et al 2005, Rehman et al 2005, Wang et al 2006. This is confirmed by both detection of GH mRNA and fluorescence studies in mice injected with GFP-expressing vector.…”

Section: Discussionsupporting

confidence: 74%

“…The choice of the dsAAV8 vectors, the dosage of the virus particles, and the i.p. route of administration were based on previous studies performed with the same viral delivery method and same promoter (McClane et al 1997, Song et al 2001, Tsai et al 2002, Ayuso et al 2004, Arruda et al 2005, Lai et al 2005, Rehman et al 2005, Wang et al 2006. The choice of HTZ animals as control is based on their normal growth pattern (Alba & Salvatori 2004), and the need to use animals with genetic background similar to the GHD mice.…”

Section: Discussionmentioning

confidence: 99%

“…Following guidance from previous studies (McClane et al 1997, Song et al 2001, Tsai et al 2002, Ayuso et al 2004, Arruda et al 2005, Lai et al 2005, Rehman et al 2005, Wang et al 2006), dsAAV8-CMV-GH viruses were injected intraperitoneally at the 10th day of age. Treatment groups are described in Table 1.…”

Section: Animals and Vector Administrationmentioning

confidence: 99%

“…The vectors carrying a foreign gene can infect both dividing and non-dividing cells in vitro and in vivo, obtaining a long-term transgenic expression minimizing the toxicity and cellular immune response (Ferrari et al 1996, Afione et al 1999, Bangari & Mittal 2006, resulting in robust and long-term delivery to many tissues, including muscle , liver (Song et al 2001), and pancreas (McClane et al 1997, Ayuso et al 2004. These dsAAV vectors have successfully delivered gene therapy in animal models of diabetes (McClane et al 1997, Ayuso et al 2004, Rehman et al 2005, Wang et al 2006, muscular dystrophy , hemophilia (Arruda et al 2005), loss of corneal endothelium (Lai et al 2005), and cerebral ischemia (Tsai et al 2002), with possible important future applications in humans.…”

Section: Introductionmentioning

confidence: 99%

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A single injection of double-stranded adeno-associated viral vector expressing GH normalizes growth in GH-deficient mice

Sagazio

Xiao

Wang

et al. 2007

Journal of Endocrinology

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GH is secreted by the somatotropic cells of the pituitary gland, and its deficiency (GHD) impairs longitudinal growth. Due to its short half-life, GH therapy needs administration of GH injections daily. Adeno-associated viral vectors (AAV) can deliver gene therapy to animals with possible future applications in humans. The new generation of double-stranded AAV vectors (dsAAV) provides widespread, strong, and stable transgene expression without toxicity and immune response. To determine whether such a new system could be used to deliver GH to a mouse model of isolated GHD due to ablation of the GHRH knock-out gene (GHRHKO), we have created AAV viral particles containing mouse GH cDNA driven by a cytomegalovirus promoter (dsAAV8-CMV-GH), and tested them in male GHRHKO mice. GHRHKO animals received either a single (low dose) or two (high dose) i.p. injections of dsAAV8-CMV-GH (1!10 11 particles) at the 10th and 11th days of age, or a placebo injection, and were followed up to the 6th or 24th week of life. A single dsAAV8-GH injection caused body length and weight normalization. At week 6, serum GH was higher in mice receiving both virus doses compared with controls, while it was normal at week 24. Serum IGF-1 increased in both virus-treated groups, and it was normal at 24 weeks. GH mRNA expression was detected in liver, skeletal, and heart muscle of virus-injected animals. These data show that normalization of longitudinal growth can be reached in GHD mice using a single injection of a doublestranded adeno-associated virus expressing GH.

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Section: Discussionsupporting

confidence: 74%

Section: Discussionmentioning

confidence: 99%

Section: Animals and Vector Administrationmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

A single injection of double-stranded adeno-associated viral vector expressing GH normalizes growth in GH-deficient mice

Sagazio

Xiao

Wang

et al. 2007

Journal of Endocrinology

Self Cite

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“…12 Ex vivo transduction by incubation of corneal explants in the presence of different types of viral vectors has also been shown to lead to gene transfer to the corneal endothelium. [13][14][15][16] Nevertheless, only one of these studies reports an efficient transduction of the keratocytes, but this was achieved only after several days of incubation of corneal organ culture with adeno-associated virus vectors. 16 However, transduction of the keratocytes of the corneal stroma may have a strong impact by controlling the local environment.…”

mentioning

confidence: 99%

Efficient lentiviral gene transfer into corneal stroma cells using a femtosecond laser

2009

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We investigated a new procedure for gene transfer into the stroma of pig cornea for the delivery of therapeutic factors. A delimited space was created at 110 mm depth with a LDV femtosecond laser in pig corneas, and a HIV1-derived lentiviral vector expressing green fluorescent protein (GFP) (LV-CMV-GFP) was injected into the pocket. Corneas were subsequently dissected and kept in culture as explants. After 5 days, histological analysis of the explants revealed that the corneal pockets had closed and that the gene transfer procedure was efficient over the whole pocket area. Almost all the keratocytes were transduced in this area. Vector diffusion at right angles to the pocket's plane encompasses four (endothelium side) to 10 (epithelium side) layers of keratocytes. After 21 days, the level of transduction was similar to the results obtained after 5 days. The femtosecond laser technique allows a reliable injection and diffusion of lentiviral vectors to efficiently transduce stromal cells in a delimited area. Showing the efficacy of this procedure in vivo could represent an important step toward treatment or prevention of recurrent angiogenesis of the corneal stroma.

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Nanoneedles Induce Targeted siRNA Silencing of p16 in the Human Corneal Endothelium

et al. 2022

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Nanoneedles can target nucleic acid transfection to primary cells at tissue interfaces with high efficiency and minimal perturbation. The corneal endothelium is an ideal target for nanoneedle‐mediated RNA interference therapy aimed at enhancing its proliferative capacity, necessary for tissue regeneration. This work develops a strategy for siRNA nanoninjection to the human corneal endothelium. Nanoneedles can deliver p16‐targeting siRNA to primary human corneal endothelial cells in vitro without toxicity. The nanoinjection of siRNA induces p16 silencing and increases cell proliferation, as monitored by ki67 expression. Furthermore, siRNA nanoinjection targeting the human corneal endothelium is nontoxic ex vivo, and silences p16 in transfected cells. These data indicate that nanoinjection can support targeted RNA interference therapy for the treatment of endothelial corneal dysfunction.

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Highly EfficientEx VivoGene Delivery into Human Corneal Endothelial Cells by Recombinant Adeno-Associated Virus

Cited by 19 publications

References 23 publications

A single injection of double-stranded adeno-associated viral vector expressing GH normalizes growth in GH-deficient mice

A single injection of double-stranded adeno-associated viral vector expressing GH normalizes growth in GH-deficient mice

Efficient lentiviral gene transfer into corneal stroma cells using a femtosecond laser

Nanoneedles Induce Targeted siRNA Silencing of p16 in the Human Corneal Endothelium

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