Research
Interests
Dr. Samulski's
research focuses on the study of the dependent parvovirus adeno-associated
virus. AAV is the only known DNA animal virus which requires co-infection
by a second unrelated virus in order to undergo productive infection.
Dr. Samulski
has cloned the AAV genome into the bacterial plasmid pBR322 and
demonstrated that this recombinant clone is infectious when introduced
into human cells co-infected with a helper virus. This recombinant
clone has provided a manipulatable system for the analysis of
mechanism(s) involved in excision and integration of the adeno-associated
virus genome. Based on these observations, he has been able to
test AAV as a alternative viral vector for gene delivery. The
ability to generate non-pathogenic viral vectors for current basic
research have the long term potential of serving as reagents for
use in clinical settings. He has established successful and long
term gene expression over a year, which directly addresses the
issue of molecular therapy required for genetic disorders. One
of Dr. Samulski's current goals of research is to continue to
derive delivery systems for use in gene therapy (content
from Dr.
Samulski's Pharmacology Web page).
Other Resources
Dr.
Samulski @ UNC Dept. of Pharmacology
Selected
Publications
Click
here for a list of publications from PubMed
Flierl
A, Chen Y, Coskun PE, Samulski RJ, Wallace DC. Adeno-associated
virus-mediated gene transfer of the heart/muscle adenine nucleotide
translocator (ANT) in mouse. Gene Ther. 2005 Jan 13
McCarty
DM, Young SM, Samulski RJ. Integration
of Adeno-associated virus (AAV) and recombinant AAV vectors.
Annu Rev Genet. 2004 Dec;38:819-845.
Stilwell
JL, Samulski RJ. Role
of viral vectors and virion shells in cellular gene expression.
Mol Ther. 2004 Mar;9(3):337-46.
Fu
H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X, McCarty
DM. Self-complementary
adeno-associated virus serotype 2 vector: global distribution
and broad dispersion of AAV-mediated transgene expression in mouse
brain. Mol Ther. 2003 Dec;8(6):911-7.
Stilwell
JL, Samulski RJ. Adeno-associated
virus vectors for therapeutic gene transfer. Biotechniques.
2003 Jan;34(1):148-50, 152, 154 passim.
Weitzman
MD, Young SM Jr, Cathomen T, Samulski RJ. Targeted
integration by adeno-associated virus. Methods Mol Med.
2003;76:201-19.
Haberman
R, Criswell H, Snowdy S, Ming Z, Breese G, Samulski R, McCown
T. Therapeutic
liabilities of in vivo viral vector tropism: adeno-associated
virus vectors, NMDAR1 antisense, and focal seizure sensitivity.
Mol Ther. 2002 Oct;6(4):495-500.
Wheeler,
MD, Kono, H, Yin, M, Nakagami, M, Uesugi, T, Arteel, GE, Gabele,
E, Rusyn, I, Yamashina, S, Froh, M, Adachi, Y, Iimuro, Y, Gbradford,
BU, Smutney, OM, Connor, HD, Mason, RP, Goyert, SM, Peters, JM,
Gonzales, FJ, Samulski, RJ, Thurman, RG. The
Role of Kupffer cell oxidant production in early ethanol-induced
liver disease. Free Radic Biol Med. 2001 Dec 15; 31
(12) 1544-9. Review.
Fu,
H, Samulski, RJ, McCown, TJ, Picornell, YJ, Fletcher, D, Muenzer,
J. Neurological
Correction of Lysosomal Storage in a Mucopolysaccharidosi IIB
Mouse Model by Adeno-associated Virus-Mediated Gene Delivery.
Moleculary Therapy January 2002, vol 5 #1, 42-49.
Rabinowitz,
JE, Rolling, F, Li, C, Conrath, HervE, C, Xiao, W, Xiao, X, Samulski,
RJ. Cross
Packaging of a Single AAV Type 2 Vector Genome into Multiple AAV
Serotypes Enables Transduction with Broad Specifity. Journal
of Virology, January 2002, vol 76 #2.
Rabinowitz,
JE, Samulski, RJ. Looking into the Eclipse. Molecular Therapy
Vol. 5, March 2002.
Janson,
C, McPhee, S, Bilaniuk, L, Haselgrove, J, Testaiuti, M, Freese,
A, Wang, DJ, Shera, D, Hurh, P, Rupin, J, Saslow, E, Goldfarb,
O, Goldberg, M, Larijani, G, Sharrar, W, Liouterman, L, Camp,
A, Kolodny, E, Samulski, J, Leone, P. Gene therapy of Canavan
Disease AAV-2 Vector for Neurosurgical Delivery of Aspartoacylase
Gene (ASPA) to the Human Brain. Clinical Protocol. Human Gene
Therapy 131391-1412, July 20, 2002.
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