Showing posts with label gene therapy. Show all posts
Showing posts with label gene therapy. Show all posts
Thursday, September 21, 2023

Gene Therapy For Sickle Cell Disease

›
Here is a paper from several years ago, abstract: Sickle cell disease results from a homozygous missense mutation in the β-globin gene ...
Thursday, September 15, 2022

Genome Editing In The (Mouse) Brain

›
Here is a paper from several years ago discussing a fascinating and very promising finding.  We'll need to take a look at any subseque...
Thursday, May 19, 2022

Gene Editing For Muscular Dystrophy

›
Here is a paper from several years ago discussing promising results with gene therapy in a mouse model of muscular dystrophy.  Abstract: ...
Thursday, October 21, 2021

Gene Therapy For Hearing Loss: Vector Development

›
One major problem for gene therapy is designing a safe and effector vector for delivering the gene therapy material to the target cells.tis...
Friday, September 21, 2018

More On CRISPR

›
Using new methodologies to optimize the CRISPR system .  Abstract: CRISPR-Cas systems offer versatile technologies for genome engineer...
Wednesday, August 1, 2018

Multiplex Genome Editing

›
It is now becoming possible to perform multiple editing of genomes , a "DNA construction site" toolkit, which can enable a num...
Saturday, June 30, 2018

CRISPR Editing In Blood Stem Cells

›
CRISPR editing in blood stem cells ; abstract: Genome editing via homologous recombination (HR) (gene targeting) in human hematopoieti...
Monday, June 25, 2018

p53 Inhibits CRISPR-Cas9 Engineering In Human Pluripotent Stem Cells

›
Human pluripotent stem cells are difficult to engineer with the CRSPR system compared to other cell types, which is unfortunate given the g...
Wednesday, May 23, 2018

Another CRISPR Modification

›
Customizing cell circuits for gene expression control.  Abstract: The catalytically dead Cpf1 endonuclease from Acidaminococcus sp. BV...
Monday, May 21, 2018

Gene Therapy For Beta-Thalassemia

›
Successful gene therapy ; abstract: BACKGROUND: Donor availability and transplantation-related risks limit the broad use of allogene...
Tuesday, May 1, 2018

Gene Therapy Delivery

›
Public Domain, https://commons.wikimedia.org/w/index.php?curid=488192 By Nandiyanto - Own work, Public Domain, http...
Tuesday, April 17, 2018

DNA Nanorobots Against Cancer

›
DNA nanorobots against cancer. From article: DNA nanorobots are a somewhat new concept for drug delivery. They work by getting ...
Sunday, March 11, 2018

Hemophilia Gene Therapy Success

›
Advances in hemophilia gene therapy ; abstract: BACKGROUND: The prevention of bleeding with adequately sustained levels of clotting ...
Sunday, February 4, 2018

Epigenetic CRISPR

›
Epigenetics is the modulation of gene expression through modification of chromatin (e.g., by methylation and/or acetylation) without chan...
Wednesday, January 31, 2018

THOR And Cancer

›
THOR may promote cancer , not Thor the Asgardian god, but THOR the long non-coding RNA. Targeting THOR via gene therapy may be a novel an...
Sunday, January 28, 2018

CRISPR Targeting RNA

›
The CRISPR system is a crucially important, relatively new, system for targeted modification of DNA, and one that has implications for gene...
Tuesday, January 16, 2018

Essential Genes And Cancer Immunotherapy

›
Here is a paper looking at what genes may be essential for successful cancer immunotherapy. Abstract: Somatic gene mutations can alter...
Tuesday, December 26, 2017

High-Fidelity CRISPR

›
A more accurate “high-fidelity” CRISPR system will enable more precise editing of genomes and opens up the hope for optimized gene therapy...
Sunday, December 24, 2017

Gene Therapy For Deafness

›
Here we see a CRISPR-based system for gene therapy against a form of hereditary deafness, tested fairly successfully in the Beethoven mou...
Wednesday, December 20, 2017

Looping DNA

›
The formation of DNA loops is an integral mechanism in the control of gene expression, bringing various control regions of DNA, and their...