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How one cell transplant platform is poised to tackle complex diseases
Lineage Cell Therapeutics CEO Brian Culley discusses the company's cell therapy technology with
. The technology involves the transplantation of differentiated stem cells as part of an off-the-shelf allogeneic cell therapy.
New tool sheds light on transient protein interactions in cells
Researchers have developed a novel chemical tool for elucidating protein interaction networks within cells. The technique, details of which were published in
on April 23, allows for the visualization of protein-protein interactions.
A CRISPR on-off switch for genes controls expression without altering DNA
A new gene silencing tool allows scientists to switch genes on and off without altering genetic sequences. The tool, described in a paper published in
on April 9, uses a modified CRISPR-Cas9 system to introduce reversible epigenetic changes that control gene expression.
New gene therapy could be effective in treating complex polygenic conditions
Scientists are applying gene therapy approaches in a new way by simultaneously administering a combination of cargos to treat complex polygenic neurodegenerative diseases with no single genetic cause. Details of the combination gene therapy in two animal models were detailed in a March 31
Gene therapy based on transcription factors could be effective against Alzheimer's
A type of gene therapy using transcription factors to target DNA has been shown to dramatically reduce levels of the harmful protein tau in preclinical mouse models of Alzheimer's disease. The findings, published in
on March 19, suggest that the therapy could easily translate to the clinic and be effective in humans.
Customized gene therapies successfully target rare eye diseases
Can gene therapies prove effective for the treatment of rare genetic diseases? One company may have found a solution that incorporates a high degree of customization, manufacturing expertise, and years' worth of knowledge.
Imaging finds tooth decay's root, may lead to ways to fight it
Atom probe tomography and other techniques revealed that human enamel contains small chemical flaws that may affect the resiliency of the fundamental building blocks of teeth, in a study published July 2 in
Extracellular RNA identifies new Alzheimer's biomarker
The association of extracellular RNA with upregulation of the phosphoglycerate dehydrogenase gene may be a useful presymptomatic biomarker for Alzheimer's disease, according to an article published in
on March 26.
New machinery digests toxic proteins to protect DNA replication
Researchers have identified molecular machinery that helps repair damaged DNA that occurs during DNA replication and transcription. This work, published in
on March 9, reveals how specific enzymes "eat" or break down proteins that cause broken DNA.
Glia-to-neuron conversion gene therapy can treat Huntington's disease
Using adeno-associated virus technology, researchers have developed a novel gene therapy that can regenerate functional neurons in mouse models of Huntington's disease. The work was published in
on February 27.
American Society of Clinical Oncology (ASCO) Annual Meeting
European Association for Cancer Research (EACR) Virtual Congress
European Society of Human Genetics (ESHG) 2021
Pharma R&D Week
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Interline raises $92M for drug discovery of dysfunctional proteins
Biogen, Envisagenics collaborate on RNA splicing
Flare launches with $82M investment
Rare Disease Company Coalition established
EBRC explains new ethics in engineering research statement
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