An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells.
Jae Hyung Jang,James T. Koerber,Jung Suk Kim,Prashanth Asuri,Tandis Vazin,Melissa A. Bartel,Albert J. Keung,Inchan Kwon,Kook In Park,David V. Schaffer +9 more
TLDR
This work applied directed evolution to create a "designer" AAV vector with enhanced delivery efficiency for neural stem cells (NSCs), and a novel AAV variant, carrying an insertion of a selected peptide sequence on the surface of the threefold spike within the heparin-binding site, emerged from this evolution.About:
This article is published in Molecular Therapy.The article was published on 2011-04-01 and is currently open access. It has received 108 citations till now. The article focuses on the topics: Gene delivery & Adeno-associated virus.read more
Citations
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Journal ArticleDOI
A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons.
D. Gowanlock R. Tervo,Bum Yeol Hwang,Sarada Viswanathan,Thomas Gaj,Maria Lavzin,Maria Lavzin,Kimberly D. Ritola,Sarah Lindo,Susan Michael,Elena Kuleshova,Elena Kuleshova,David Stephen Ojala,Cheng Chiu Huang,Charles R. Gerfen,Charles R. Gerfen,Jackie Schiller,Jackie Schiller,Joshua T. Dudman,Adam W. Hantman,Loren L. Looger,David V. Schaffer,Alla Y. Karpova +21 more
TL;DR: A newly evolved variant of adeno-associated virus, rAAV2-retro, permits robust retrograde access to projection neurons with efficiency comparable to classical synthetic retrograde tracers and enables sufficient sensor/effector expression for functional circuit interrogation and in vivo genome editing in targeted neuronal populations.
Journal ArticleDOI
Engineering adeno-associated viruses for clinical gene therapy
TL;DR: New approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome barriers to extension of clinical gene therapy successes to many other human diseases.
Journal ArticleDOI
The AAV Vector Toolkit: Poised at the Clinical Crossroads
TL;DR: The discovery of naturally occurring adeno-associated virus isolates in different animal species and the generation of engineered AAV strains using molecular genetics tools have yielded a versatile AAV vector toolkit and a battery of second generation AAV vectors are now available.
Journal ArticleDOI
Viral Vectors for Gene Therapy: Translational and Clinical Outlook
TL;DR: This review highlights key historical trends in clinical gene therapy, the recent clinical successes of viral-based gene Therapy, and current research that may enable future clinical application.
Journal ArticleDOI
Targeting protein and peptide therapeutics to the heart via tannic acid modification.
Mikyung Shin,Hyang Ae Lee,Mihyun Lee,Yoomi Shin,Ji-Joon Song,Sun-Woong Kang,Dae Hwan Nam,Eun Je Jeon,Mira Cho,Minjae Do,Sun-Hyun Park,Moon Sue Lee,Jae Hyung Jang,Seung Woo Cho,Ki-Suk Kim,Haeshin Lee +15 more
TL;DR: It is shown that the modification of protein and peptide therapeutics with tannic acid improves their ability to specifically target heart tissue, as shown with a rat model of myocardial ischaemia-reperfusion injury.
References
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Journal ArticleDOI
Rapid evolution of a protein in vitro by DNA shuffling.
TL;DR: It is reported here that selected mutants had a minimum inhibitory concentration of 640 μg ml-1, a 32,000-fold increase and 64-fold greater than any published TEM-1 derived enzyme.
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Progress and problems with the use of viral vectors for gene therapy
TL;DR: With the development of a leukaemia-like syndrome in two patients cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
Journal ArticleDOI
Germline Transmission and Tissue-Specific Expression of Transgenes Delivered by Lentiviral Vectors
TL;DR: Transgenic mice carrying the green fluorescent protein (GFP) gene driven by a ubiquitously expressing promoter are generated and transgenic rats that express GFP at high levels are generated, suggesting that this technique can be used to produce other transgenic animal species.
Journal ArticleDOI
Highly efficient endogenous human gene correction using designed zinc-finger nucleases
Fyodor D. Urnov,Jeffrey C. Miller,Ya-Li Lee,Christian Beauséjour,Jeremy M. Rock,Sheldon Augustus,Andrew C. Jamieson,Matthew H. Porteus,Philip D. Gregory,Michael C. Holmes +9 more
TL;DR: It is shown that zinc-finger nucleases designed against an X-linked severe combined immune deficiency mutation in the IL2Rγ gene yielded more than 18% gene-modified human cells without selection, raising the possibility of strategies based on zinc- finger nucleases for the treatment of disease.
Journal ArticleDOI
The development of neural stem cells
TL;DR: A rush of papers proclaiming adult stem cell plasticity has fostered the notion that there is essentially one stem cell type that, with the right impetus, can create whatever progeny the authors' heart, liver or other vital organ desires, but studies aimed at understanding the role of stem cells during development have led to a different view — that stem cells are restricted regionally and temporally, and thus not all stem cell types are equivalent.
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