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Category: genetics – Page 18

Digit regeneration in mice is stimulated by sequential treatment with FGF2 and BMP2

Basically whole body regeneration is definitely possible we just need to right genetic code to push the regeneration button in the human body much like how these mice had their digits regenerated so too we can regenerate just like Deadpool or even the axolotl.

Wound fibrosis after amputation in mammals is replaced with regeneration of amputated structural elements by sequential FGF2/BMP2 treatment. Regenerated tissues include phalangeal/sesamoid bones, tendon/ligament, synovial joint, articular cartilage.

From fragment to form: wholebody regeneration in a model urochordate Medicine

Whole body regeneration is possible just would need to find it in human beings similar genetics.

Rinkevich, Y., Rinkevich, B. From fragment to form: whole-body regeneration in a model urochordate. npj Regen Med 10, 36 (2025). https://doi.org/10.1038/s41536-025-00423-0

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What Can We Learn From the Most Genetically Modified Human Alive? | Liz Parrish

In 2015, Liz Parrish flew to Colombia and let someone inject an untested gene therapy into her body 150 times. She texted her kids that she loved them before it started. When it was over, she went for nachos.

Ten years later, her telomeres are longer than when she started, and she has taken 12 gene therapies in total.

Her decade of self-experimentation has produced peer-reviewed data, a growing protocol of gene therapies, and a company training its sights on making biological aging optional. Our conversation goes over what it took to become patient zero and what gene therapy for aging looks like in practice today, among other things.

3D DNA mapping in rare immune cells reveals new genes linked with autoimmune disease risk

Our DNA is often pictured as a simple spiral, like a piece of rope held taut. But inside cells, it folds into a complex three-dimensional structure, with 2 meters (6.6 feet) of DNA scrunched like a headphone wire in a pocket. This bundled architecture plays a crucial role in how genes are switched on and off. Understanding these interactions is key to interpreting genetic studies of disease and can help develop targeted treatments.

Genes—the instructions for making proteins—are regulated by two types of DNA regions, called promoters and enhancers. Promoters are located at the start of each gene and directly load the machinery that reads it to produce RNA, a template for making proteins. In contrast, enhancers, which act like “molecular switches” boosting gene activity in the right cell and condition, may be found much further from the genes they control: sometimes many thousands or even millions of DNA letters away. When DNA folds in the 3D space of the cell’s nucleus, these enhancers loop around to physically contact the genes they regulate.

Understanding how enhancers work and which genes they control is particularly important because genome-wide association studies—large-scale efforts comparing the DNA of thousands of people to identify small genetic differences linked to disease—have revealed many genetic variants associated with complex conditions such as Crohn’s disease that sit within these regions. Crohn’s disease is a common form of inflammatory bowel disease, which affects around 1 in 100 people globally and currently has no known cure.

Elucidating the spatiotemporal dynamics of glucose metabolism with genetically encoded fluorescent biosensors

Recent years have seen the rapid development of genetically encoded fluorescent sensors that can achieve specific and sensitive monitoring of metabolites in glucose metabolism. In this perspective, Li et al. discuss the different biosensors and their applications in understanding glucose metabolism.

MicroRNA-based gene therapy to treat ALS

A single IV injection of a microRNA-based biologic suppressed production of the mutant SOD1 protein that causes amyotrophic lateral sclerosis (ALS); delayed disease onset by 60 days; and extended lifespan by 100 days, more than triple the average survival time, in mice models of the disease.

The gene therapy, delivered via adeno-associated virus (AAV) vector, preserved motor neurons and maintained neuromuscular connections in treated animals, which translated into improved muscle and respiratory function, motor performance, and lifespan in pre-clinical studies. These findings, published in Nature Communications, have the potential for clinical application in patients with SOD1-caused ALS, as well as other neurodegenerative diseases caused by toxic, gain-of-function gene mutations.

“These therapeutic benefits, from a single IV injection, are unprecedented among gene therapy approaches in this mouse model,” said the senior author. “No other studies have been able to achieve this kind of survival extension. This makes us very optimistic that our approach could have a meaningful impact for patients suffering from this horrible disease and warrants further clinical evaluation.”

Overcoming Cancer Resistance: Strategies and Modalities for Effective Treatment

Resistance to cancer drugs is a complex phenomenon that poses a significant challenge in the treatment of various malignancies. This review comprehensively explores cancer resistance mechanisms and discusses emerging strategies and modalities to overcome this obstacle. Many factors contribute to cancer resistance, including genetic mutations, activation of alternative signaling pathways, and alterations in the tumor microenvironment. Innovative approaches, such as targeted protein degradation, immunotherapy combinations, precision medicine, and novel drug delivery systems, hold promise for improving treatment outcomes. Understanding the intricacies of cancer resistance and leveraging innovative modalities are essential for advancing cancer therapy.

Gene Therapy Improves Stem Cell Transplant Success

Stem cell transplantation (or bone marrow transplantation) and gene therapy have revolutionized the way oncologists treat patients. Both approaches have the potential to cure patients with sickle cell disease, b-thalassemia, immune disorders, and even several blood cancers. There are specific transplants that can occur: autologous and allogenic. Both forms of transplantation are dependent on the source of the donor cells. Autologous transplants use the patient’s healthy stem cells taken from the patient before systemic treatment, like chemotherapy. Allogenic transplants use stem cells collected from healthy donors. Typically stems cells for an allogenic transplant is from a family member or umbilical cord blood to genetically match the recipient. If stem cells are not genetically matched, then the donor will recognize the stem cells as foreign and reject the transplant. This rejection is known as Graft-versus-Host Disease (GvHD). Infusion of stem cells only takes about 15 minutes, but the engraftment or the ability for cells to proliferate and establish themselves in the bone marrow takes up to 18 days. For full immune system recovery, most physicians see patients recover within a year. However, stem cell transplants are not perfect. There is a high rate of GvHD, infection, and disease relapse. Patients also usually receive chemotherapy before a transplant, which increases toxicity and reduces success of the procedure. Scientists are currently working on how to improve the success rate of transplantation.

A recent article in Nature, by Dr. Pietro Genovese and others, demonstrated that stem cell transplantations are safer when chemotherapy is replaced with a targeted treatment. Researchers used antibodies that recognize and target markers on blood-forming stem cells. This approach helps clear harmful preexisting stem cells from the patient before transplantation, instead of using toxic chemotherapeutic agents that damage DNA throughout the body. The antibody approach is more specific and reduces toxicity in patients. Genovese is an Assistant Professor at Dana Farber and Boston Children’s Cancer and Blood Disorders Center. His work focuses on gene-editing and bioengineering technologies that improve stem and immune cells. Specifically, Genovese investigates ways to improve gene therapy and develop approaches to enhance treatment for patients with hematological malignancies.

Previously, an antibody could not discern between current recipient and infused autologous stem cells. Antibodies could also attack transplanted cells from a separate donor (allogenic). To avoid these issues, Genovese and his team have used gene-editing tools to select for a specific biomarker on the surface of donor stem cells. The small edit prevented the antibody from binding to the donor stem cells. These antibodies were able to avoid the healthy stem cells and still eliminate the infected stem cells. This approach allows the donor stem cells to properly graft in the host and improve success of transplantation.