Lifeboat Foundation

LONDON (AP) — Britain’s medicines regulator has authorized the world’s first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. In a statement Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020. The agency approved…

The one-time treatment helped relieve symptoms of disease and could free patients from the need for bone marrow transplants or regular blood transfusions, Beach said, adding that the drug hopefully offers a permanent fix for the condition.

The MHRA said it identified no significant safety concerns during the trials and will continue to closely monitor Casgevy’s safety after approval.

Vertex CEO and President Reshma Kewalramanit celebrated Casgevy’s approval as “a historic day in science and medicine” and Samarth Kulkarni, CEO and Chairman of Crispr Therapeutics, said it will hopefully mark “the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases.” The two companies are hoping for similarly positive decisions from the MHRA’s counterparts in the Europe Union and the U.S., which are in the process of evaluating Casgevy, also known as exa-cel. The Food and Drug Administration is expected to make a decision in early December and has a deadline of December 8. The agency appears poised to follow the MHRA and approve the treatment, with its advisors confident of the drug’s efficacy and benefit but wary of theoretical unintended consequences of genetic modifications.

Summary: Researchers made a breakthrough in memory research by genetically modifying the LIMK1 protein, crucial for memory, to be controlled by the drug rapamycin.

This study demonstrates the ability to enhance memory functions by manipulating synaptic plasticity in the brain.

The engineered protein showed significant memory improvement in animal models with age-related cognitive decline, offering potential for innovative treatments for neuropsychiatric diseases like dementia. This ‘chemogenetic’ approach, blending genetics and chemistry, opens new avenues in neurological research and therapy.

At Oak Ridge National Laboratory (ORNL), quantum biology, artificial intelligence, and bioengineering have collided to redefine the landscape of CRISPR Cas9 genome editing tools. This multidisciplinary approach, detailed in the journal Nucleic Acids Research, promises to elevate the precision and efficiency of genetic modifications in organisms, particularly microbes, paving the way for enhanced production of renewable fuels and chemicals.

CRISPR is adept at modifying genetic code to enhance an organism’s performance or correct mutations. CRISPR Cas9 requires a guide RNA (gRNA) to direct the enzyme to its target site to perform these modifications. However, existing computational models for predicting effective guide RNAs in CRISPR tools have shown limited efficiency when applied to microbes. ORNL’s Synthetic Biology group, led by Carrie Eckert, observed these disparities and set out to bridge the gap.

“A lot of the CRISPR tools have been developed for mammalian cells, fruit flies, or other model species. Few have been geared towards microbes where the chromosomal structures and sizes are very different,” explained Eckert.

The AI tools were complemented by quantum biology and bioengineering approaches.

Philip Gray/ORNL, U.S. Dept. of Energy.

Combining several advances.

Continue reading “AI improves genome editing of microbes to produce renewable fuels” »

Adoptive cell therapy has emerged as a promising alternative treatment for hematological and solid cancers, with CAR-T therapy standing out as a prominent avenue. In this approach, T cells are genetically engineered with chimeric antigen receptors (CARs) to enhance their targeting capabilities^1–2. The outcome of CAR-T cell therapy hinges on a complex interplay of phenotype, activation, and functional profiling of these engineered cells. Immunophenotypic characterization of CAR-T cells assumes a pivotal role in ensuring treatment quality and facilitating continuous monitoring of treatment response¹. In the process of immunophenotyping, engineered T cells are separated based on their markers to characterize the composition of the cell population within the sample. The strategic identification and isolation of specific CAR-T cell subsets is essential in augmenting therapy responses².

Deciphering Cellular Composition, Defining CAR-T Therapy Efficacy

Immunophenotyping is a pivotal technique that combines specific antibodies with fluorescent compounds to reveal specific protein expression in cell populations to identify categorize the tagged cells. Immunophenotyping leverages the differences in surface markers among T cells, reflecting their differentiation, activation, and memory status². These markers provide insights into immune cell development, function, proliferation potential, and long-term viability. The distinct surface marker profiles closely correlate with the efficacy of CAR-T cell therapy³. Essential markers for immunophenotypic analysis, including CD3, CD4, CD8, CD45RA, CD34R0, CCR7, CD27, and CD95, are presented in Table 1.

So they volunteered for an experimental cholesterol-lowering treatment using gene editing that was unlike anything tried in patients before.

The result, reported Sunday by the company Verve Therapeutics of Boston at a meeting of the American Heart Association, showed that the treatment appeared to reduce cholesterol levels markedly in patients and that it appeared to be safe.

The trial involved only 10 patients, with an average age of 54. Each had a genetic abnormality, familial hypercholesterolemia, that affects around one million people in the United States. But the findings could also point the way for millions of other patients around the world who are contending with heart disease, which remains a leading cause of death. In the United States alone, more than 800,000 people have heart attacks each year.

New research finds RNA

Ribonucleic acid (RNA) is a polymeric molecule similar to DNA that is essential in various biological roles in coding, decoding, regulation and expression of genes. Both are nucleic acids, but unlike DNA, RNA is single-stranded. An RNA strand has a backbone made of alternating sugar (ribose) and phosphate groups. Attached to each sugar is one of four bases—adenine (A), uracil (U), cytosine ©, or guanine (G). Different types of RNA exist in the cell: messenger RNA (mRNA), ribosomal RNA (rRNA), and transfer RNA (tRNA).

Oak Ridge National Laboratory’s research in quantum biology and AI has significantly improved the efficiency of CRISPR Cas9 genome editing in microbes, aiding in renewable energy development.

Scientists at Oak Ridge National Laboratory (ORNL) used their expertise in quantum biology, artificial intelligence, and bioengineering to improve how CRISPR Cas9 genome editing tools work on organisms like microbes that can be modified to produce renewable fuels and chemicals.

CRISPR is a powerful tool for bioengineering, used to modify genetic code to improve an organism’s performance or to correct mutations. The CRISPR Cas9 tool relies on a single, unique guide RNA.