Curing Diseases With CRISPR

-
Over the past several years, huge advances in genome editing technologies have fueled an almost palpable excitement about the future of genome engineering. Beginning with the discovery of zinc-finger nucleases, and followed recently by the description of TALEN and CRISPR genome-editing systems, the possibility of literally rewriting a genome has quickly gone from dream to reality.

An incredibly exciting potential use of genome-editing technologies is to correct genetic mutations that cause diseases. Everyone aware of CRISPR and TALEN technology has obviously considered this possibility, and the medical and biotechnology fields are working towards the development of the first genome editing-based treatments.

Now, for the first time, researchers have demonstrated that CRISPR can be used to eliminate a disease-causing mutation from cells in an animal model of a human disease. These three studies, published in the most recent issue of Science, show that the CRISPR system can effectively rewrite the genome of mice carrying a genetic defect causing muscular dystrophy. They demonstrated that editing can be done in cell culture or directly in an animal, to effectively cure the disease.

It now seems like only a matter of time before genome-editing is applied to human patients, and hopes are high for the medical potential of CRISPR and TALEN.

Bibliography

  1. Tabebordbar M, Zhu K, Cheng JK, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ. (2016) In vivo gene editing in dystrophic mouse muscle and muscle stem cells. Science 351:407-11.
  2. Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA. (2016) In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy. Science 351:403-7. 
  3. Long C, Amoasii L, Mireault AA, McAnally JR, Li H, Sanchez-Ortiz E, Bhattacharyya S, Shelton JM, Bassel-Duby R, Olson EN. (2016) Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Science 351:400-3.
Original Source: Cyagen Biosciences

Comments

Post a Comment

Popular posts from this blog

Cells driving gecko's ability to re-grow its tail identified

-
Image
A U of G researcher is the first to discover the type of stem cell that is behind the gecko's ability to re-grow its tail, a finding that has implications for spinal cord treatment in humans. Many lizards can detach a portion of their tail to avoid a predator and then regenerate a new one. Unlike mammals, the lizard tail includes a spinal cord. Prof. Matthew Vickaryous found that the spinal cord of the tail contained a large number of stem cells and proteins known to support stem cell growth. "We knew the gecko's spinal cord could regenerate, but we didn't know which cells were playing a key role," said Vickaryous, lead author of the study recently published in the  Journal of Comparative Neurology . "Humans are notoriously bad at dealing with spinal cord injuries so I'm hoping we can use what we learn from geckos to coax human spinal cord injuries into repairing themselves." Geckos are able to re-grow a new tail within 30 days -- faste
Read more

Knockout by CRISPR vs Knockdown by shRNA

-
In solving the mystery of gene function, there is no more important clue than the phenotype of inactivating the gene of interest. With a plethora of methods available, researchers must first determine what approach is best for their specific scientific questions and experimental systems. For over a decade, RNA-interference-based methods of gene knockdown (i.e. RNAi & shRNA) have provided a wealth of insight into gene function, but in recent years the advent of CRISPR- and TALEN-based methods now allow genome editing to be used to quickly and efficiently test the effect of gene knockouts. Here, we review the advantages and disadvantages of these approaches, and describe some experimental situations in which one approach is better than another, focusing primarily on CRISPR/Cas9 and shRNA. For a discussion of the relative advantages of CRISPR/Cas9 versus TALEN , see our prior Newsletter on this topic. CRISPR Knockout In this method, a guide RNA (gRNA) homologous to an 18-22nt ta
Read more

Beyond ACE2: Additional Targets of Significance for Coronavirus Research

-
At present, the prevention and treatment of coronavirus is the most urgent issue for scientific and medical research. In addition to the ACE2 host receptor target that is being highly focused on, what other targets deserve the attention of current coronavirus researchers? Herein, experts from Cyagen investigate the lesser-known targets that are pertinent to coronavirus research, aiming to provide some helpful references to guide scientific researchers. 1. DPP4 Dipeptidyl peptidase (DPP4) belongs to the family of proline-specific serine proteases and is capable of cleaving dipeptides from the amino terminal (N-terminal). DPP4 is the most typical peptidase in this family, and it is also the third exo-peptidase that was found to be a coronavirus receptor (after ACE2 and APN). DPP4 is a multifunctional type II transmembrane glycoprotein with 766 amino acids; it exists as a homodimer on the cell surface. The dimerization of peptidase depends on the molecules’ connection between the
Read more