Current Gene Therapy

Thursday, November 24, 2016

Free Trial Request

Bentham Science Publishers3:22 AM No comments

Free Trial Request

Call For Paper

Bentham Science Publishers2:38 AM No comments

Bentham Science Publishers would like to invite you to submit your research paper for publishing in the Journal of
Current Gene Therapy

Highlighted Article: Seq and You Will Find

Bentham Science Publishers11:06 PM No comments

Seq and You Will Find

[ Vol. 16 , Issue. 3 ]

Author(s):

Nicole Schonrock, Nicky Jonkhout and John S. Mattick Pages 220 - 229 ( 10 )

Abstract:

The human genome sequence is freely available, nearly complete and is providing a foundation of research opportunities that are overturning our current understanding of human biology. The advent of next generation sequencing has revolutionized the way we can interrogate the genome and its transcriptional products and how we analyze, diagnose, monitor and even treat human disease. Personal genetic profiles are increasing dramatically in medical value as researchers accumulate more and more knowledge about the interaction between genetic and environmental factors that contribute to the onset of common disorders. As the cost of sequencing plummets, whole genome sequencing of individuals is becoming a reality and the field of personalized genomic medicine is rapidly developing. Now there is great need for accurate annotation of all functionally important sequences in the human genome and the variations within them that contribute to health and disease. The vast majority of our genome gives rise to RNA transcripts. This extraordinarily versatile molecule not only encodes protein information but also has great structural dynamics and plasticity, capacity for DNA/RNA/protein interactions and catalytic activity. It is a key regulator of biological networks with clear links to human disease and a more comprehensive understanding of its function is needed to maximise its use in medical practice. This review focuses on the complexity of our genome and the impact of sequencing technologies in understanding its many products and functions in health and disease.

Keywords:

Next generation sequencing, Non-coding RNA, GWAS, Personalized medicine, Capture-sequencing.

Affiliation:

Garvan Institute of Medical Research, Darlinghurst, 2010, NSW, Australia.

Read Full-Text article

For More Information Please Visit Our Website Current Gene Therapy

Most Accessed Article: AAV Vectorization of DSB-mediated Gene Editing Technologies

Bentham Science Publishers2:59 AM No comments

AAV Vectorization of DSB-mediated Gene Editing Technologies

Author(s):
Rachel J. Moser and Matthew L. HirschPages 207-219 (13)

Abstract:

Recent work both at the bench and the bedside demonstrate zinc-finger nucleases (ZFNs), CRISPR/Cas9, and other programmable site-specific endonuclease technologies are being successfully utilized within and alongside AAV vectors to induce therapeutically relevant levels of directed gene editing within the human chromosome. Studies from past decades acknowledge that AAV vector genomes are enhanced substrates for homology-directed repair in the presence or absence of targeted DNA damage within the host genome. Additionally, AAV vectors are currently the most efficient format for in vivo gene delivery with no vector related complications in >100 clinical trials for diverse diseases. At the same time, advancements in the design of custom-engineered site-specific endonucleases and the utilization of elucidated endonuclease formats have resulted in efficient and facile genetic engineering for basic science and for clinical therapies. AAV vectors and gene editing technologies are an obvious marriage, using AAV for the delivery of repair substrate and/or a gene encoding a designer endonuclease; however, while efficient delivery and enhanced gene targeting by vector genomes are advantageous, other attributes of AAV vectors are less desirable for gene editing technologies. This review summarizes the various roles that AAV vectors play in gene editing technologies and provides insight into its trending applications for the treatment of genetic diseases.

Keywords:

AAV Vectorization, DSB, CRISPR/Cas9, DNA, ZFNs.

Affiliation:
Department of Ophthalmology, Gene Therapy Center, University of North Carolina at Chapel Hill, Campus Box 7352, Chapel Hill, NC 27599-7352, USA.

Read Full-Text article

For More Information Please Visit Our Website Current Gene Therapy