Bioengineering

Scientists rehabilitate disease-causing T cells into tolerance builders

Tue, 04 Nov 2025 08:00:00 -0500

For 75 years, the standard tools for autoimmune disease have consisted of steroids, cytotoxics and broad biologics that tamp down the entire immune system. They can help, but they are rarely curative. “They’re blunt instruments,” Regcell Inc. CEO Mike McCullar told BioWorld. “They can’t distinguish good immune cells and bad immune cells,” which is why many carry black-box warnings and must be taken for years, sometimes for life.

Scientists rehabilitate disease-causing T cells into tolerance builders

Mon, 03 Nov 2025 08:00:00 -0500

Epigenetic switch and gene editing activate human T cells

Fri, 24 Oct 2025 09:00:00 -0400

Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.

Epigenetic switch and gene editing activate human T cells

Wed, 22 Oct 2025 09:00:00 -0400

Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco (UCSF) have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.

Epigenetic approach shows durable PCSK9 silencing in primates

Wed, 15 Oct 2025 09:00:00 -0400

A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.

At ESGCT, emerging technologies for in vivo therapies

Thu, 09 Oct 2025 09:00:00 -0400

The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

ESGCT 2025: Redefining CAR T cells across cancer and autoimmunity

Wed, 08 Oct 2025 09:00:00 -0400

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

CRISPR boosts CAR T cells for leukemia and myeloma

Tue, 30 Sep 2025 09:00:00 -0400

Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.

ASGCT 2025: Overcoming kidney complexity in gene and cell therapy

Fri, 16 May 2025 09:00:00 -0400

Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.

New mouse model of atherosclerosis improves upon previous designs

Mon, 23 Sep 2024 09:00:00 -0400

Scientists from the Cardiovascular Research Center at the University of Virginia School of Medicine and Astrazeneca plc have developed a new mouse model of cardiovascular disease associated with genetic variations of cholesterol metabolism. The animal allows in vivo studies of myocardial infarction, plaque rupture and stroke.

Epigenetic editor silences prion protein gene in the brain

Mon, 22 Jul 2024 09:00:00 -0400

Epigenetic silencing could prevent the production of proteins that cause pathologies. CHARM (coupled histone tail for autoinhibition release of methyltransferase), a DNA methylation-based editor, suppressed transcription of prion proteins in the brains of mice.

A gene therapy could restore hearing in adults

Fri, 12 Jul 2024 09:00:00 -0400

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

A gene therapy could restore hearing in adults

Thu, 11 Jul 2024 09:00:00 -0400

Engineered plasma cells produce effective bispecific antibodies against leukemia

Fri, 05 Jul 2024 12:00:00 -0400

Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.

Engineered plasma cells produce effective bispecific antibodies against leukemia

Thu, 04 Jul 2024 09:00:00 -0400

“We are trying to engineer plasma cells to make as a stable source for biologic drugs. One thing that is really unique about plasma cells is that they can live for a really long time … up to 10 years or even 100 years depending on the type of plasma cell that that you make,” Richard James, senior author of the study, principal investigator at Seattle Children’s Research Institute, and associate professor at the University of Washington, told BioWorld.

New techniques open the way for large-scale programmable genome editing

Tue, 02 Jul 2024 11:00:00 -0400

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

New techniques open the way for large-scale programmable genome editing

Thu, 27 Jun 2024 11:00:00 -0400

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.

New techniques open the way for large-scale programmable genome editing

Wed, 26 Jun 2024 11:00:00 -0400

New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications.

A pan-approach against blood cancer preserving hematopoiesis

Wed, 29 May 2024 09:00:00 -0400

A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.

Next-generation genome editing tools surpass CRISPR milestone

Thu, 04 Jan 2024 12:00:00 -0500

Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease. But science does not stop.

Next-generation genome editing tools surpass CRISPR milestone

Thu, 28 Dec 2023 09:00:00 -0500

Giving yeast cells aging indecision increases longevity

Thu, 04 May 2023 09:00:00 -0400

Cells of Saccharomyces cerevisiae, a yeast used as a model for human mitosis, age in two ways. Both genomic instability and the decline of mitochondria cause cells to degenerate and die. The choice of one type or another depends on a network of genes that can be adjusted by bioengineering.

Gene editing restores vision in mice with retinitis pigmentosa

Thu, 23 Mar 2023 09:00:00 -0400

A modification of the CRISPR technique has made it possible to restore vision in mouse models with retinitis pigmentosa (RP). Scientists at the Institute of Visual Neuroscience and Stem Cell Engineering of Wuhan University of Science and Technology developed a new gene-editing tool called PE(SpRY) to edit in vivo a mutation of enzyme phosphodiesterase 6B (PDE6β) and return its function.

Synthetic cell junctions allow tissue reconstruction

Tue, 13 Dec 2022 09:00:00 -0500

A combination of bioengineering techniques on normal cell binding proteins could be the method of the future for selective cell binding. Scientists at the University of California, San Francisco (UCSF) have created a synthetic glue based on the expression of membrane receptors to establish the desired connection between cells. The results may be applied in different fields of cell biology or biomedicine, such as regeneration and wound repair, including the nervous system, or cancer.

Human brain organoids mature after transplantation into rats

Fri, 14 Oct 2022 09:00:00 -0400

Human brain organoids transplanted into rats could be used as an in vivo model for the study of neuropsychiatric diseases. Researchers at Stanford University managed to mature human organoid neurons in the somatosensory cortex of the animal's brain and incorporate them into its neural circuitry.The integration improved the morphological and physiological properties of the transplanted neurons. Compared to those of organoids in a Petri dish, human cells preserved their own identity, and they modified the rat's learned behavior through stimulation and reward experiments.

Human brain organoids mature after transplantation into rats

Thu, 13 Oct 2022 09:00:00 -0400