Lifeboat Foundation: Safeguarding Humanity
Toggle light / dark theme

Blog

Category: biotech/medical – Page 1,155

The BACE1 enzyme has a rate-limiting role in the amyloidogenic pathway (see Glossary) and has been extensively studied for its neuronal functions[1]. Since 2000, intensive efforts have focused on developing small-molecule BACE1 inhibitors to reduce amyloid β (Aβ) production in Alzheimer’s disease (AD) brains. However, human clinical trials involving most BACE1 inhibitors were stopped at Phase 2/3 due to limited therapeutic benefits[2]. BACE1 inhibitors act by reducing Aβ-related pathologies in AD brains, that is, they are used to treat the symptoms rather than the underlying disease.

Read more | >

Huntington’s disease (HD) is a neurological disorder that causes progressive loss of movement, coordination and cognitive function. It is caused by a mutation in a single gene called huntingtin (HTT). More than 200,000 people worldwide live with the genetic condition, approximately 30,000 in the United States. More than a quarter of a million Americans are at risk of inheriting HD from an affected parent. There is no cure.

But in a new study, published December 12, 2022 in Nature Neuroscience, researchers at University of California San Diego School of Medicine, with colleagues elsewhere, describe using RNA-targeting CRISPR/Cas13D technology to develop a new therapeutic strategy that specifically eliminates toxic RNA that causes HD.

CRISPR is known as a genome-editing tool that allows scientists to add, remove or alter genetic material at specific locations in the genome. It is based on a naturally occurring immune defense system used by bacteria. However, current strategies run the risk of off-target edits at unintended sites that may cause permanent and inheritable chromosomal insertions or genome alterations. Because of this, significant efforts have focused on identifying CRISPR systems that target RNA directly without altering the genome.

Read more | >

An international research team led by Dr. Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior of the cell.

Patients with mutations in this protein suffer from Allan-Herndon-Dudley syndrome, a that takes the form of serious neurological alterations, in which each patient may reveal a different mutation of MCT8.

This study, published in Neurobiology of Disease, describes the first avatar model for the disease—in other words, the first animal model with the same as various .

Read more | >

Summary: Researchers engineered cells containing customized adhesion molecules that bind to specific cell partners in predictable ways to form complex multicellular entities. The discovery is a major step toward building new tissue and organs.

Source: UCSF

Researchers at UC San Francisco (UCSF) have engineered molecules that act like “cellular glue,” allowing them to direct in precise fashion how cells bond with each other. The discovery represents a major step toward building tissues and organs, a long-sought goal of regenerative medicine.

Read more | >

Summary: Researchers have developed a new compound, dubbed FexD, that can prevent and reverse inflammation in mouse models of inflammatory bowel disease.

Source: Salk Institute.

A drug developed by Salk Institute researchers acts like a master reset switch in the intestines. The compound, called FexD, has previously been found to lower cholesterol, burn fat, and ward off colorectal cancer in mice.

Read more | >

Achieving a long-sought goal of regenerative medicine.

Researchers from the University of California, San Francisco, (UCSF) engineered molecules that function as “cellular glue,” enabling them to precisely direct how cells bond with each other. This is a significant step toward regenerative medicine’s long-term goal of creating new tissues and organs, according to a press release.

Adhesive molecules are naturally present in the body and keep the tens of trillions of cells together in organized patterns. They build neural networks, develop structures, and direct immune cells to specific areas of the body. Adhesion also makes cell communication easier to maintain the body functioning as a self-regulating whole.

Read more | >

𝐂𝐀𝐑-𝐓-𝐜𝐞𝐥𝐥 𝐬𝐡𝐨𝐰𝐬 𝐩𝐫𝐨𝐦𝐢𝐬𝐞 𝐢𝐧 𝐩𝐚𝐭𝐢𝐞𝐧𝐭𝐬 𝐰𝐢𝐭𝐡 𝐥𝐲𝐦𝐩𝐡𝐨𝐦𝐚 𝐨𝐟 𝐭𝐡𝐞 𝐛𝐫𝐚𝐢𝐧 𝐚𝐧𝐝 𝐬𝐩𝐢𝐧𝐚𝐥 𝐜𝐨𝐫𝐝 𝐢𝐧 𝐞𝐚𝐫𝐥𝐲 𝐭𝐫𝐢𝐚𝐥

𝘼 𝘾𝘼𝙍-𝙏-𝙘𝙚𝙡𝙡 𝙩𝙝𝙚𝙧𝙖𝙥𝙮 𝙠𝙣𝙤𝙬𝙣 𝙖𝙨 𝙖𝙭𝙞𝙘𝙖𝙗𝙩𝙖𝙜𝙚𝙣𝙚 𝙘𝙞𝙡𝙤𝙡𝙚𝙪𝙘𝙚𝙡 (𝙖𝙭𝙞-𝙘𝙚𝙡) 𝙞𝙨 𝙨𝙖𝙛𝙚 𝙖𝙣𝙙 𝙨𝙝𝙤𝙬𝙨 𝙚𝙣𝙘𝙤𝙪𝙧𝙖𝙜𝙞𝙣𝙜 𝙨𝙞𝙜𝙣𝙨 𝙤𝙛 𝙚𝙛𝙛𝙞𝙘𝙖𝙘𝙮 𝙞𝙣 𝙖 𝙨𝙢𝙖𝙡𝙡 𝙥𝙞𝙡𝙤𝙩 𝙩𝙧𝙞𝙖𝙡 𝙞𝙣𝙫𝙤𝙡𝙫𝙞𝙣𝙜 𝙥𝙖𝙩𝙞𝙚𝙣𝙩𝙨 𝙬𝙞𝙩𝙝 𝙡𝙮𝙢𝙥𝙝𝙤𝙢𝙖 𝙤𝙛 𝙩𝙝𝙚 𝙗𝙧𝙖𝙞𝙣 𝙖𝙣𝙙/𝙤𝙧 𝙨𝙥𝙞𝙣𝙖𝙡 𝙘𝙤𝙧𝙙, 𝘿𝙖𝙣𝙖-𝙁𝙖𝙧𝙗𝙚𝙧 𝘾𝙖𝙣𝙘𝙚𝙧 𝙄𝙣𝙨𝙩𝙞𝙩𝙪𝙩𝙚 𝙞𝙣𝙫𝙚𝙨𝙩𝙞𝙜𝙖𝙩𝙤𝙧𝙨 𝙧𝙚𝙥𝙤𝙧𝙩 𝙖𝙩 𝙩𝙝𝙚 64𝙩𝙝 𝘼𝙢𝙚𝙧𝙞𝙘𝙖𝙣 𝙎𝙤𝙘𝙞𝙚𝙩𝙮 𝙤𝙛 𝙃𝙚𝙢𝙖𝙩𝙤𝙡𝙤𝙜𝙮 (𝘼𝙎𝙃) 𝘼𝙣𝙣𝙪𝙖𝙡 𝙈𝙚𝙚𝙩𝙞𝙣𝙜.

A CAR-T-cell therapy known as axicabtagene ciloleucel (axi-cel) is safe and shows encouraging signs of efficacy in a small pilot trial involving patients with lymphoma of the brain and/or spinal cord, Dana-Farber Cancer Institute investigators report at the 64th American Society of Hematology (ASH) Annual Meeting.

The research features an in-depth, molecular study of individual CAR-T cells isolated from patients’ blood and cerebrospinal fluid (CSF), which surrounds the brain and spinal cord. This unprecedented analysis, conducted in collaboration with the Cellular Therapeutics and Systems Immunology Lab (CTSI), directed by Leslie Kean, MD, PhD, at Dana-Farber and Boston Children’s Hospital, reveals a surprising difference between the two CAR-T-cell populations: the cells in the CSF display a molecular signature that indicates activation of the interferon pathway, an important step in rallying the immune system. These studies are reported in two oral abstracts at ASH. “For many patients with lymphoma of the central nervous system, there aren’t great treatment options,” said Dana-Farber’s Caron Jacobson, MD, MMSc, who led the trial and will present the findings at ASH. “Our early results suggest that expanding the applicability of CAR-T cells to this indication could improve patient outcomes.”

Lymphomas can begin within the brain or spinal cord, or the tumors can spread to those sites (known collectively as the central nervous system or CNS) after they originate in other parts of the body. While the underlying biology of these primary and secondary CNS lymphomas can be quite different, these cancers are often difficult to treat, especially once the tumors evade standard treatments. In that case, patients typically do not live more than 2 years.

Read more | >

Would you want to take one home if it freed you up from the hassles of pregnancy?

The concept of the world’s first-ever artificial womb facility has been unveiled. Called EctoLife, the facility can produce up to 30,000 babies every year. The use of the word “produce” is deliberate since, as one can see in the video below, the facility aims to give parents a wide range of tools to get a baby customized to their desires.

Hashem Al-Ghaili/ YouTube.

The concept and need for such a facility are pretty clear. The global human population is now reaching its peak and is expected to fall from here. The world’s richest person, Elon Musk, has been vocal about this and, by fathering nine children so far, believes he is doing his best to prevent the world from suffering a population collapse.

Read more | >

Playing through the greenery and litter of a mini forest’s undergrowth for just one month may be enough to change a child’s immune system, according to an experiment in Finland.

When daycare workers rolled out a lawn, planted forest undergrowth (such as dwarf heather and blueberries), and allowed children to care for crops in planter boxes, the diversity of microbes in the guts and on the skin of the young kids appeared healthier in a very short space of time.

Compared to other city kids who play in standard urban daycares with yards of pavement, tile, and gravel, 3-, 4-, and 5-year-olds at these greened-up daycare centers in Finland showed increased T-cells and other important immune markers in their blood within 28 days.

Read more | >