Richard Jude Samulski

Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat

Young, Samuel M., Samulski, Richard Jude

Adeno-associated virus (AAV) is the only known eukaryotic virus capable of targeted integration in human cells. An AAV Rep binding element (RBE) and terminal resolution site (trs) identical to the...

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao, Xiao, Li, Juan, Samulski, Richard Jude

Recently, efficient and long-term in vivo gene transfer by recombinant adeno-associated virus type 2 (rAAV) vectors has been demonstrated in a variety of tissues. Further improvement in vector titer...

Roles of Adeno-Associated Virus Rep Protein and Human Chromosome 19 in Site-Specific Recombination

Young, Samuel M., McCarty, Douglas M., Degtyareva, Natalya, Samulski, Richard Jude

Adeno-associated virus type 2 (AAV) is the only known eucaryotic virus capable of targeted integration in human cells. AAV integrates preferentially into human chromosome (ch) 19q13.3qter. The...

Charge-to-Alanine Mutagenesis of the Adeno-Associated Virus Type 2 Rep78/68 Proteins Yields Temperature-Sensitive and Magnesium-Dependent Variants

Gavin, Denise K., Young, Samuel M., Xiao, Weidong, Temple, Brenda, Abernathy, Corinne R., Pereira, Daniel J., ...

The adeno-associated virus type 2 (AAV) replication (Rep) proteins Rep78 and 68 (Rep78/68) exhibit a number of biochemical activities required for AAV replication, including specific binding to a...

Novel Transcriptional Regulatory Signals in the Adeno-Associated Virus Terminal Repeat A/D Junction Element

Haberman, Rebecca P., McCown, Thomas J., Samulski, Richard Jude

Adeno-associated virus (AAV) type 2 vectors transfer stable, long-term gene expression to diverse cell types in vivo. Many gene therapy applications require the control of long-term transgene...

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

Summerford, Candace, Samulski, Richard Jude

The human parvovirus adeno-associated virus (AAV) infects a broad range of cell types, including human, nonhuman primate, canine, murine, and avian. Although little is known about the initial events...

Adeno-associated virus vectors: potential applications for cancer gene therapy

Li, Chengwen, Bowles, Dawn E, Van Dyke, Terry, Samulski, Richard Jude

Augmenting cancer treatment by protein and gene delivery continues to gain momentum based on success in animal models. The primary hurdle of fully exploiting the arsenal of molecular targets and...

Adeno-associated virus (AAV) site-specific recombination does not require a Rep-dependent origin of replication within the AAV terminal repeat

Young, Samuel M., Samulski, Richard Jude

Adeno-associated virus (AAV) is the only known eukaryotic virus capable of targeted integration in human cells. An AAV Rep binding element (RBE) and terminal resolution site (trs) identical to the...

Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

Xiao, Xiao, Li, Juan, Samulski, Richard Jude

Recently, efficient and long-term in vivo gene transfer by recombinant adeno-associated virus type 2 (rAAV) vectors has been demonstrated in a variety of tissues. Further improvement in vector titer...

Roles of Adeno-Associated Virus Rep Protein and Human Chromosome 19 in Site-Specific Recombination

Young, Samuel M., McCarty, Douglas M., Degtyareva, Natalya, Samulski, Richard Jude

Adeno-associated virus type 2 (AAV) is the only known eucaryotic virus capable of targeted integration in human cells. AAV integrates preferentially into human chromosome (ch) 19q13.3qter. The...

Charge-to-Alanine Mutagenesis of the Adeno-Associated Virus Type 2 Rep78/68 Proteins Yields Temperature-Sensitive and Magnesium-Dependent Variants

Gavin, Denise K., Young, Samuel M., Xiao, Weidong, Temple, Brenda, Abernathy, Corinne R., Pereira, Daniel J., ...

The adeno-associated virus type 2 (AAV) replication (Rep) proteins Rep78 and 68 (Rep78/68) exhibit a number of biochemical activities required for AAV replication, including specific binding to a...

Novel Transcriptional Regulatory Signals in the Adeno-Associated Virus Terminal Repeat A/D Junction Element

Haberman, Rebecca P., McCown, Thomas J., Samulski, Richard Jude

Adeno-associated virus (AAV) type 2 vectors transfer stable, long-term gene expression to diverse cell types in vivo. Many gene therapy applications require the control of long-term transgene...

Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

Summerford, Candace, Samulski, Richard Jude

The human parvovirus adeno-associated virus (AAV) infects a broad range of cell types, including human, nonhuman primate, canine, murine, and avian. Although little is known about the initial events...

α2,3 and α2,6 N-Linked Sialic Acids Facilitate Efficient Binding and Transduction by Adeno-Associated Virus Types 1 and 6

Wu, Zhijian, Miller, Edward, Agbandje-McKenna, Mavis, Samulski, Richard Jude

Recombinant adeno-associated viruses (AAVs) are promising vectors in the field of gene therapy. Different AAV serotypes display distinct tissue tropism, believed to be related to the distribution of...

Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction

Wang, Jinhui, Xie, Jing, Lu, Hui, Chen, Lingxia, Hauck, Bernd, Samulski, Richard Jude, ...

Previous studies have documented that 0.1≈1% of input recombinant adeno-associated virus (rAAV) vectors could be stabilized and lead to transgene expression. To characterize the steps involving...