Lifeboat Foundation

A gene editing tool using a system known as CRISPR-Cas9 has recently been approved by the U.S. Food and Drug Administration (FDA) for sickle cell disease. The drug is known as Casgevy and the media has hailed this treatment as a ‘cure’ for sickle cell anemia patients. While it is still unclear if the drug completely cures these patients, clinical trials show exciting efficacy.

Sickle cell disease is a genetic blood disorder affecting thousands of US citizens. Many of these patients are African American and Hispanic. In sickle cell disease, hemoglobin, a protein in red blood cells that helps carry oxygen throughout the body, is mutated. As a result, blood cells change shape in the form of a sickle, giving the disease its name. Unfortunately, the mutated cells cause disruption of blood flow and prevent other blood cells from delivering oxygen to the body. This disease is extremely rare and can lower the quality of life in patients. Previously, there were limited treatments options including transfusions and medications for pain management. However, Casgevy provides a new option to help treat the patient and relieve pain for over a year after a single treatment.

One-time treatment using Casgevy improved life quality for sickle cell patients. A single-arm trial was conducted at multiple health centers in adults and adolescents. These patients were screened for two vaso-occlusive crises (VOCs) which are described as severely painful events due to a lack of oxygen delivery from sickle cell blood cells blocking blood flow. The primary measure of success in the trial was the number of VOCs after treatment. In total, 44 patients received Casgevy and 33 were able to follow up and be evaluated. Of the 33 patients that made it through the trial, 29 of them did not experience any VOCs for 12 months. This is a 93.5% success rate based on the number of patients that were analyzed. All 44 patients were able to successfully undergo treatment without any graft rejection. In addition, researchers concluded that this treatment was not only effective, but safe with few side effects.

Engineers at MIT, Penn State University, and Carnegie Mellon University have devised a way to manipulate cells in three dimensions using sound waves. These “acoustic tweezers” could make possible 3D printing of cell structures for tissue engineering and other applications, the researchers say.

Designing tissue implants that can be used to treat human disease requires precisely recreating the natural tissue architecture, but so far it has proven difficult to develop a single method that can achieve that while keeping cells viable and functional.

“The results presented in this paper provide a unique pathway to manipulate biological cells accurately and in three dimensions, without the need for any invasive contact, tagging, or biochemical labeling,” says Subra Suresh, president of Carnegie Mellon and former dean of engineering at MIT. “This approach could lead to new possibilities for research and applications in such areas as regenerative medicine, neuroscience, tissue engineering, biomanufacturing, and cancer metastasis.”

Feng Guo, an associate professor of intelligent systems engineering at the Indiana University Luddy School of Informatics, Computing and Engineering, is addressing the technical limitations of artificial intelligence computing hardware by developing a new hybrid computing system—which has been…

A team of IU bioengineers are building the intersection of brain organoids and artificial intelligence, which could potentially transform the performance and efficiency of advanced AI techniques.

Using CRISPR, an immune system bacteria use to protect themselves from viruses, scientists have harnessed the power to edit genetic information within cells. In fact, the first CRISPR-based therapeutic was recently approved by the FDA to treat sickle cell disease in December 2023. That therapy is based on a highly studied system known as the CRISPR-Cas9 genetic scissor.

However, a newer and unique platform with the potential to make large-sized DNA removals, called Type I CRISPR or CRISPR-Cas3, waits in the wings for potential therapeutic use.

A new study from Yan Zhang, Ph.D., Assistant Professor in the Department of Biological Chemistry at the University of Michigan Medical School, and her collaborators at Cornell University develops off-switches useful for improving the safety of the Type I-C/Cas3 gene editor. The study, “Exploiting Activation and Inactivation Mechanisms in Type I-C CRISPR-Cas3 for 3 Genome Editing Applications,” is published in the journal Molecular Cell.

Gene editing is revolutionizing the understanding of health and disease, providing researchers with vast opportunities to advance the development of novel treatment approaches. Traditionally, researchers used various methods to introduce double strand breaks (DSBs) into the genome, including transactivator-like effectors, meganucleases, and zinc finger nucleases. While useful, these techniques are limited in that they are time and labor intensive, less efficient, and can have unintended effects. In contrast, the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein-9 (Cas9) system (CRISPR/Cas9) is among the most sensitive and efficient methods for creating DNA DSBs, making it the leading gene editing technology.

CRISPR/Cas9 is a naturally occurring immune protective process that bacteria use to destroy foreign genetic material.¹ Researchers repurposed the CRISPR/Cas9 system for genetic engineering applications in mammalian cells, exploiting the molecular processes that introduce DSBs in specific sections of DNA, which are then repaired to turn certain genes on or off, or to correct genomic errors with extraordinary precision.^2,3 This technology’s applications are far reaching, from cell culture and animal models to translational research that focuses on correcting genetic mutations in diseases such as cancer, hemophilia, and sickle cell disease.⁴

Researchers exploit plasmids, the small, closed circular DNA strands native to bacteria, as delivery vehicles in CRISPR/Cas9 gene editing protocols. Plasmids shuttle the CRISPR/Cas9 gene editing components to target cells and can be manipulated to control gene editing activity, including targeting multiple genes at a time. Plasmids can also deliver gene repair instructions and machinery. For example, poly (ADP-ribose) polymerase 1 (PARP1) is an enzyme that drives DNA repair and transcription.⁵ It is a critical aspect of CRISPR/Cas9 gene editing technology in part because it helps repair the DSBs created by the CRISPR/Cas9 system. PARP1 CRISPR plasmids can edit, knockout, or upregulate PARP1 gene expression depending on the specific instructions encoded in the plasmid.

Bioengineered bacteria sneak past solid tumor defenses to guide CAR T cells’ attacks.

For the first time in the company’s 40-year history, Liebherr have converted one of their R 9,400 excavators from a conventional diesel to an electric powertrain.

The re-powered mining machine was commissioned at Fortescue’s Christmas Creek mine site in Western Australia – but what’s more significant is that the conversion was done during the standard service life of the machine.

“The modular design of Liebherr equipment makes it possible to repower existing diesel excavators to new zero emission configurations, such as electric powertrains,” explains Oliver Weiss, Executive Vice President of R&D, Engineering, and Manufacturing for Liebherr Mining. “This means that the diesel equipment customers buy today is also future-proofed for many years to come. The fact that we can ease the transition from traditional to decarbonized mining fleets for our customers is one of the key strategies of the Liebherr Zero Emission Mining Program.”

Treating complex diseases such as skin cancer often requires simultaneous administration of multiple anticancer drugs. The delivery of such life-saving therapeutic drugs has evolved with the rise of nanotechnology-based drug carriers. Nanoplatforms offer numerous advantages, including increased bioavailability, lowered dosages, and improved biodistribution.

Now a team of researchers, led by Professor Myoung-Hwan Park from Sahmyook University in South Korea, has developed a light-responsive nanofiber-based novel drug delivery system (DDS) targeting skin cancer. The DDS was studied in a detailed manner, beginning with its synthesis and characterization to its biocompatibility, drug release profile, and efficacy against skin cancer. These research findings are published in the Journal of Drug Delivery Science and Technology.

Explaining the motivation behind the present research, Dr. Park states, “Conventional drugs can be efficiently delivered in a controlled manner through nano-engineered platforms, and such an approach increases the overall effectiveness of the treatment. This approach improves outcomes in cancer drug therapy by ensuring precise delivery at optimal dosages.”

Tissue engineering, which involves the use of grafts or scaffolds to aid cell regeneration, is emerging as a key medical practice for treating volumetric muscle loss (VML), a condition where a significant amount of muscle tissue is lost beyond the body’s natural regenerative capacity. To improve surgical outcomes, traditional muscle grafts are giving way to artificial scaffold materials, with MXene nanoparticles (NPs) standing out as a promising option.

MXene NPs are 2D materials primarily composed of transition-metal carbides and nitride. They are highly electrically conductive, can accommodate a wide range of functional groups, and have stacked structures that promote cell interactions and muscle growth. While there have been practical demonstrations in the laboratory showcasing their ability to promote the reconstruction of skeletal muscles, the specific mechanism by which they do so remains unclear.

To address this gap, Associate Professor Yun Hak Kim from the Department of Anatomy and Department of Biomedical Informatics, alongside Professors Suck Won Hong, and Dong-Wook Han from the Department of Cogno-Mechatronics Engineering at Pusan National University developed nanofibrous matrices containing MXene NPs as scaffolds. They used DNA sequencing to reveal the genes and biological pathways activated by MXene NPs to aid in muscle regeneration.