Theresa A. Storm

Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver (2008)

Hiroyuki Nakai, Xiaolin Wu, Sally Fuess, Theresa A. Storm, David Munroe, Eugenio Montini, ...

Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low frequency of chromosomal integration of vector genomes, recent studies have raised concerns about the...

Nonrandom Transduction of Recombinant Adeno-Associated Virus Vectors in Mouse Hepatocytes In Vivo: Cell Cycling Does Not Influence Hepatocyte Transduction

Miao, Carol H., Nakai, Hiroyuki, Thompson, Arthur R., Storm, Theresa A., Chiu, Winnie, Snyder, Richard O., ...

Recombinant adeno-associated virus vectors (rAAV) show promise in preclinical trials for the treatment of genetic diseases including hemophilia. Liver-directed gene transfer results in a slow rise in...

Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombination Are Responsible for Stable Transduction of Liver In Vivo

Nakai, Hiroyuki, Storm, Theresa A., Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in experimental animals. Following portal-vein administration of rAAV vectors in vivo, single-stranded (ss) rAAV genomes...

Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

Nakai, Hiroyuki, Yant, Stephen R., Storm, Theresa A., Fuess, Sally, Meuse, Leonard, Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in experimental animals. Although the vector genomes are found both as extrachromosomes and as chromosomally integrated...

A Limited Number of Transducible Hepatocytes Restricts a Wide-Range Linear Vector Dose Response in Recombinant Adeno-Associated Virus-Mediated Liver Transduction

Nakai, Hiroyuki, Thomas, Clare E., Storm, Theresa A., Fuess, Sally, Powell, Sharon, Wright, J. Fraser, ...

Recombinant adeno-associated virus (rAAV) vectors are promising vehicles for achieving stable liver transduction in vivo. However, the mechanisms of liver transduction are not fully understood, and...

Rapid Uncoating of Vector Genomes Is the Key to Efficient Liver Transduction with Pseudotyped Adeno-Associated Virus Vectors

Thomas, Clare E., Storm, Theresa A., Huang, Zan, Kay, Mark A.

Transduction of the liver with single-stranded adeno-associated virus serotype 2 (AAV2) vectors is inefficient; less than 10% of hepatocytes are permissive for stable transduction, and transgene...

Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice

Nakai, Hiroyuki, Fuess, Sally, Storm, Theresa A., Muramatsu, Shin-ichi, Nara, Yuko, Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors can mediate long-term stable transduction in various target tissues. However, with rAAV serotype 2 (rAAV2) vectors, liver transduction is confined to...

Large-Scale Molecular Characterization of Adeno-Associated Virus Vector Integration in Mouse Liver

Nakai, Hiroyuki, Wu, Xiaolin, Fuess, Sally, Storm, Theresa A., Munroe, David, Montini, Eugenio, ...

Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low frequency of chromosomal integration of vector genomes, recent studies have raised concerns about the...

Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

Grimm, Dirk, Pandey, Kusum, Nakai, Hiroyuki, Storm, Theresa A., Kay, Mark A.

We and others have recently reported highly efficient liver gene transfer with adeno-associated virus 8 (AAV-8) pseudotypes, i.e., AAV-2 genomes packaged into AAV-8 capsids. Here we studied whether...

Nonrandom Transduction of Recombinant Adeno-Associated Virus Vectors in Mouse Hepatocytes In Vivo: Cell Cycling Does Not Influence Hepatocyte Transduction

Miao, Carol H., Nakai, Hiroyuki, Thompson, Arthur R., Storm, Theresa A., Chiu, Winnie, Snyder, Richard O., ...

Recombinant adeno-associated virus vectors (rAAV) show promise in preclinical trials for the treatment of genetic diseases including hemophilia. Liver-directed gene transfer results in a slow rise in...

Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombination Are Responsible for Stable Transduction of Liver In Vivo

Nakai, Hiroyuki, Storm, Theresa A., Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in experimental animals. Following portal-vein administration of rAAV vectors in vivo, single-stranded (ss) rAAV genomes...

Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

Nakai, Hiroyuki, Yant, Stephen R., Storm, Theresa A., Fuess, Sally, Meuse, Leonard, Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in experimental animals. Although the vector genomes are found both as extrachromosomes and as chromosomally integrated...

A Limited Number of Transducible Hepatocytes Restricts a Wide-Range Linear Vector Dose Response in Recombinant Adeno-Associated Virus-Mediated Liver Transduction

Nakai, Hiroyuki, Thomas, Clare E., Storm, Theresa A., Fuess, Sally, Powell, Sharon, Wright, J. Fraser, ...

Recombinant adeno-associated virus (rAAV) vectors are promising vehicles for achieving stable liver transduction in vivo. However, the mechanisms of liver transduction are not fully understood, and...

Rapid Uncoating of Vector Genomes Is the Key to Efficient Liver Transduction with Pseudotyped Adeno-Associated Virus Vectors

Thomas, Clare E., Storm, Theresa A., Huang, Zan, Kay, Mark A.

Transduction of the liver with single-stranded adeno-associated virus serotype 2 (AAV2) vectors is inefficient; less than 10% of hepatocytes are permissive for stable transduction, and transgene...

Unrestricted Hepatocyte Transduction with Adeno-Associated Virus Serotype 8 Vectors in Mice

Nakai, Hiroyuki, Fuess, Sally, Storm, Theresa A., Muramatsu, Shin-ichi, Nara, Yuko, Kay, Mark A.

Recombinant adeno-associated virus (rAAV) vectors can mediate long-term stable transduction in various target tissues. However, with rAAV serotype 2 (rAAV2) vectors, liver transduction is confined to...

Large-Scale Molecular Characterization of Adeno-Associated Virus Vector Integration in Mouse Liver

Nakai, Hiroyuki, Wu, Xiaolin, Fuess, Sally, Storm, Theresa A., Munroe, David, Montini, Eugenio, ...

Recombinant adeno-associated virus (rAAV) vector holds promise for gene therapy. Despite a low frequency of chromosomal integration of vector genomes, recent studies have raised concerns about the...

Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype

Grimm, Dirk, Pandey, Kusum, Nakai, Hiroyuki, Storm, Theresa A., Kay, Mark A.

We and others have recently reported highly efficient liver gene transfer with adeno-associated virus 8 (AAV-8) pseudotypes, i.e., AAV-2 genomes packaged into AAV-8 capsids. Here we studied whether...

DNA Palindromes with a Modest Arm Length of ≳20 Base Pairs Are a Significant Target for Recombinant Adeno-Associated Virus Vector Integration in the Liver, Muscles, and Heart in Mice▿

Inagaki, Katsuya, Lewis, Susanna M., Wu, Xiaolin, Ma, Congrong, Munroe, David J., Fuess, Sally, ...

Our previous study has shown that recombinant adeno-associated virus (rAAV) vector integrates preferentially in genes, near transcription start sites and CpG islands in mouse liver (H. Nakai, X. Wu,...

The Role of DNA-PKcs and Artemis in Opening Viral DNA Hairpin Termini in Various Tissues in Mice▿

Inagaki, Katsuya, Ma, Congrong, Storm, Theresa A., Kay, Mark A., Nakai, Hiroyuki

A subset of cellular DNA hairpins at double-strand breaks is processed by DNA-dependent protein kinase catalytic subunit (DNA-PKcs)- and Artemis-associated endonuclease. DNA hairpin termini of...

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses ▿ †

Grimm, Dirk, Lee, Joyce S., Wang, Lora, Desai, Tushar, Akache, Bassel, Storm, Theresa A., ...

Adeno-associated virus (AAV) serotypes differ broadly in transduction efficacies and tissue tropisms and thus hold enormous potential as vectors for human gene therapy. In reality, however, their use...

Silencing of Hepatic Fatty Acid Transporter Protein 5 in Vivo Reverses Diet-induced Non-alcoholic Fatty Liver Disease and Improves Hyperglycemia*S⃞

Doege, Holger, Grimm, Dirk, Falcon, Alaric, Tsang, Bernice, Storm, Theresa A., Xu, Hui, ...

Non-alcoholic fatty liver disease is a serious health problem linked to obesity and type 2 diabetes. To investigate the biological outcome and therapeutic potential of hepatic fatty acid uptake...