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Category: bioengineering

(Philadelphia, PA) – A single injection of a novel CRISPR gene-editing treatment safely and efficiently removes SIV – a virus related to the AIDS-causing agent HIV – from the genomes of non-human primates, scientists at the Lewis Katz School of Medicine at Temple University now report. The groundbreaking work complements previous experiments as the basis for the first-ever clinical trial of an HIV gene-editing technology in human patients, which was authorized by the Food and Drug Administration (FDA) in 2022.

The preclinical study, published online in the journal Gene Therapy, tested EBT-001, an SIV-specific CRISPR-Cas9 gene-editing therapy, in rhesus macaques. The study shows that EBT-001 effectively excises SIV from reservoirs – cells and tissues where viruses like SIV and HIV integrate into host DNA and hide for years – without any detectable off-target effects in animals. The work is a significant advance in the generation of a cure for HIV/AIDS in humans.

“Our study supports safety and demonstrates evidence of in vivo SIV editing of a CRISPR gene-editing technology aimed at the permanent inactivation of virus in a broad range of tissues in a large, preclinical animal model, using a one-time injection of the treatment,” said Kamel Khalili, PhD, Laura H. Carnell Professor and Chair of the Department of Microbiology, Immunology, and Inflammation, Director of the Center for Neurovirology and Gene Editing, Director of the Comprehensive NeuroAIDS Center at the Lewis Katz School of Medicine, and senior investigator on the new study.

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A clinical study of artificial red blood cells that can be stored for transfusions in times of emergency will begin in Japan by next March, according to Nara Medical University.

The university aims to put the artificial cells into practical use by around 2030, it said in early July, in what would likely be a world first.

The development of the blood cells, designed for use in remote areas and disasters, comes as a blood shortage is expected at medical facilities due to a declining number of donors amid the country’s shrinking population.

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Life on Earth possesses an exceptional ability to self-reproduce, which, even on a simple cellular level, is driven by complex biochemistry. But can self-reproduction exist in a biochemistry-free environment?

A study by researchers from Harvard University demonstrated that the answer is yes.

The researchers designed a non-biochemical system in which synthetic cell-like structures form and self-reproduce by ejecting polymeric spores.

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A new, easily adopted, 3D-printed device will enable scientists to create models of human tissue with even greater control and complexity. An interdisciplinary group of researchers at the University of Washington and UW Medicine led the development of the device.

3D engineering, which recently has undergone other major advances in speed and accuracy, helps design and test therapies for a range of diseases.

One goal of tissue engineering is to create lab-made environments that recreate the natural habitats of cells.

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Scientists say they’ve put together a new kind of molecular toolkit that could eventually be used to treat a variety of brain diseases, possibly including epilepsy, sleep disorders and Huntington’s disease.

The kit currently contains more than 1,000 tools of a type known as enhancer AAV vectors, with AAV standing for “adeno-associated virus.” A consortium that included researchers from Seattle’s Allen Institute for Brain Science and the University of Washington combined harmless adeno-associated viruses with snippets of engineered DNA to create a gene-therapy package that could target specific neurons in the brain while having no effect on other cells.

Researchers laid out their findings in a set of eight studies published today in the Cell Press family of journals. The work is part of a project called the Armamentarium for Precision Brain Cell Access, funded through the National Institutes of Health’s BRAIN Initiative.

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Recent technological advances are fueling the development of cutting-edge technologies that can monitor and control physiological processes with high precision. These include devices that could control the expression of genes within living organisms, without requiring invasive surgeries or procedures.

Researchers at ETH Zurich recently introduced a new method that enables the electromagnetic programming of the wireless expression regulation (EMPOWER) of transgenes in mammals, via the interfacing of and cells.

Their proposed approach, outlined in a paper published in Nature Nanotechnology, could help to treat , including diabetes, while also opening new possibilities for research in synthetic biology and regenerative medicine.

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The development of COVID-19 vaccines has sparked widespread interest. mRNA-based therapies are rapidly gaining attention owing to their unique advantages in quickly developing vaccines and immunotherapy for various ailments [1, 2]. Given that most human diseases stem from genetic factors, gene therapy represents a promising modality for addressing various inherited or acquired disorders by replacing faulty genes or silencing genes [3]. Gene therapy encompasses the targeted exploitation of genetic material, which includes gene replacement through DNA or mRNA [4, 5]; gene silencing utilizing siRNA or miRNA [6], and CRISPR-Cas9 based gene editing [7].

However, achieving safe and efficient gene delivery to specific cells requires overcoming multiple biological barriers, including extracellular obstacles such as enzyme degradation, serum protein interactions, electrostatic repulsion of genes and cell membranes, and innate immune system, as well as intracellular obstacles such as endosomal escape, transport barriers, precise release [8]. Therefore, gene vectors require several characteristics such as high gene condensation; favorable serum stability to avoid non-specific serum protein interactions, endonuclease degradation, and renal clearance; achieved specific targeting cell or tissues; effective transport into the cytoplasm thereby facilitating gene transfection (mRNA, siRNA and miRNA); precise gene release and scheduling, and nuclear localization that enables DNA transcription. Comprehensive exploration of transfection mechanisms can aid in the development of high-performance gene vectors [9, 10].

Gene vectors generally include viral vectors and non-viral vectors. Presently, approximately 70% of clinical gene therapy trials employ viral vectors, which include retroviruses, lentiviruses, adenoviruses, and adeno-associated viruses. Due to their exceptional infectivity, virus-based vectors typically exhibit excellent gene transfection capabilities. However, the clinical safety of viral vectors has been questioned due to their propensity to stimulate immunogenic reactions and induce transgene insertion mutations. Moreover, viral vectors possess several limitations, including low gene loading capacity, inability to deliver large-sized genes, complicated preparation procedures, and the patient cannot be repeatedly administered [4]. In contrast, non-viral vectors, particularly lipid nanoparticles (LNPs) and cationic polymers, have demonstrated robust gene loading capacity, heigh safety and practicability, simplicity preparation [10, 11]. Consequently, non-viral vectors are exhibiting tremendous potential for further clinical development and application. Our review primarily highlights the significant potential of non-viral vectors, particularly lipid nanoparticles (LNPs), highly branched poly(β-amino ester) (HPAE), single-chain cyclic polymer (SCKP), poly(amidoamine) (PAMAM) dendrimers, and polyethyleneimine (PEI). We intend to provide a detailed examination of the latest research progress and existing limitations of non-viral gene vectors over recent years.

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