Ex vivo autologous hematopoietic stem cell (HSC) gene therapy has provided new therapies for the treatment of hematological disorders. However, these therapies have several limitations owing to the manufacturing complexities and toxicity resulting from required conditioning regimens. Here, we developed a c-kit (CD117) antibody-targeted lipid nanoparticle (LNP) that, following a single intravenous injection, can deliver RNA (both siRNA and mRNA) to HSCs in vivo in rodents. This targeted delivery system does not require stem cell harvest, culture, or mobilization of HSCs to facilitate delivery. We also show that delivery of Cre recombinase mRNA at a dose of 1 mg kg–1 can facilitate gene editing to almost all (∼90%) hematopoietic stem and progenitor cells (HSPCs) in vivo, and edited cells retain their stemness and functionality to generate high levels of edited mature immune cells.

Venter redrew the boundaries of biology — sequencing DNA at unprecedented speed, engineering synthetic life and charting ocean microbes.

Analyses of liver biopsies from a child with spinal muscular atrophy treated with adeno-associated virus gene therapy who developed hepatitis reveal contaminating manufacturing plasmids and disrupted ...

This content is also available on my website: https://logancollinsblog.com/2026/01/02/amygdala-structure-function-and-clinically-relevant-pathways/

This study shows that non-invasive ultrasound to the human nucleus accumbens can modulate deep brain activity and enhance reward-guided learning, offering a potential alternative to invasive neuromodu...

Through the programmable self-assembly of lipid-inspired radially symmetric DNA, porous molecular membranes and cell-sized compartments are formed with applications in bottom-up biology and soft robot...

Today UNESCO’s Member States took the final step towards adopting the first global normative framework on the ethics of neurotechnology. The Recommendation, which will enter into force on November 12

Protein homeostasis, or proteostasis, is essential for cellular proteins to function properly. The buildup of abnormal proteins (such as damaged, misfolded, or aggregated proteins) is associated with ...

Of the many trends people chase in biotech, the only one that proves sure and consistent is declining returns. Even after adjusting for inflation, the number of new drugs approved per $1 billion of R&...

Targeted in situ sequencing directly maps editing rates at single-cell resolution in postmortem tissues to visualize base and prime editor delivery.

By 2030, leading AI labs will need data centers so massive they will require the power equivalent of some of America’s largest cities. Will they be able to find it?

My seventh installment of interesting research papers that I have read over the past few weeks and would like to share with my community.

Note: this content is also available on my website at https://logancollinsblog.com/2025/11/04/an-introduction-to-ebolavirus-biology/

My sixth installment of interesting research papers that I have read over the past few weeks and would like to share with my community.

Here, Audrey Chagnot and Axel Montagne discuss the structural and functional features of the blood–brain barrier and the association of its disruption with various pathologies.

We here introduce a novel bioreducible polymer-based gene delivery platform enabling widespread transgene expression in multiple brain regions with therapeutic relevance following intracranial convection-enhanced delivery. Our bioreducible nanoparticles provide markedly enhanced gene delivery efficacy in vitro and in vivo compared to nonbiodegradable nanoparticles primarily due to the ability to release gene payloads preferentially inside cells. Remarkably, our platform exhibits competitive gene delivery efficacy in a neuron-rich brain region compared to a viral vector under previous and current clinical investigations with demonstrated positive outcomes. Thus, our platform may serve as an attractive alternative for the intracranial gene therapy of neurological disorders.

With the growing threat of antibiotic resistance, unconventional approaches to antimicrobial discovery are needed. Here, the authors present a peptide topology that mimics virus architecture and assembles into antimicrobial capsids that disrupt bacterial membranes upon contact.

Note: this content is also available on my website at https://logancollinsblog.com/2025/05/16/notes-on-ultrasound-physics/

In brief, Santos-Pujol and colleagues characterize the multiomics landscape of the human with the most extreme longevity. The study enables the association of advantageous genetic variants, an engaged...

My fifth installment of interesting research papers that I have read over the past few weeks and would like to share with my community.

We introduce a two-pronged strategy comprising focused ultrasound (FUS)-mediated blood–brain barrier (BBB) opening and long-circulating biodegradable nanoparticles (NPs) for systemic delivery of nucle...

Lithium has an essential role in the brain and is deficient early in Alzheimer’s disease, which can be recapitulated in mice and treated with a novel lithium salt that restores the physiological level.

The complexity of vocal communication in some primates, whales and birds might approach that of human language.

Intravascular electroencephalography (ivEEG) using micro-intravascular electrodes was developed. Cortical-vein ivEEG showed a higher signal-to-noise ratio and finer spatial resolution of somatosensor....

By combining bioorthogonal metabolic labelling and resolution enhancement through sequential imaging of DNA barcodes, the molecular organization of individual sugars in the native glycocalyx has been ...

JUPITER, the European Union’s new exascale supercomputer, is 100% powered by renewable energy. What will researchers use it for?

Climate change, changing migration patterns, and our agriculture system have created a perfect environment for fungal pathogens to thrive.

A new suggestion that complexity increases over time, not just in living organisms but in the nonliving world, promises to rewrite notions of time and evolution.

My fourth installment of interesting research papers that I have read over the past few weeks and would like to share with my community.

Fluorescent dyes that are bright, stable, small, and biocompatible are needed for high-sensitivity two-photon imaging, but the combination of these traits has been elusive. We identified a class of sq...

A detailed conformational analysis of N-substituted bispidines has been performed to determine the factors governing the restricted rotational motion induced by the substituents. This investigation co...

Receptor-mediated transcytosis across the blood–brain barrier (BBB) may be a useful way to transport therapeutics into the brain. Here we report th...

CAPPSID relies on an engineered S. typhimurium to act as a synthetic ‘capsid’ to transcribe and deliver viral RNA inside cancer cells, launching a virus that can directly lyse surrounding cells.

Amyloid-related imaging abnormalities (ARIA), side effects of anti-amyloid drugs seen in magnetic resonance imaging of the brain, are a major safety concern in patients with Alzheimer’s disease. We de...

Prelude This essay is intended as an intuitive examination of a reward system neural circuit which may serve as a useful target for new therapies aimed at fighting treatment-resistant depression (T…

Personal site for posts about my interests: the biotech industry, medicine, molecular biology, neuroscience, biorisk, science, consciousness, AI, innovation, decision making, philosophy, games, sci-fi...

Microscopy and genomics are used to characterize cell function, but approaches to connect the two types of information are lacking, particularly at subnuclear resolution. Here, we describe expansion i...

“No matter who you are, most of the smartest people work for someone else.” - Bill Joy (co-founder of Sun Microsystems)

The mechanisms underlying the effects of ultrasonic modulation of neural activity are unclear. Here, the authors show that focused ultrasound excites cultured primary murine cortical neurons via calci...

The foundations of modern medicine rest upon 2 revolutionary changes in medical practice. The first is the development of effective treatments that have transformed previously fatal diseases into mana...

After finding the homeschooling life confining, the teen petitioned her way into a graduate class at Berkeley, where she ended up disproving a 40-year-old conjecture.

In natural habitats, nutrient availability limits bacterial growth. We discovered that bacteria can overcome this limitation by acquiring nutrients by lysing neighboring cells through contact-dependen...

High-density electrode arrays such as Neuropixels are transformative for neuroscience research, but optimized surgical and experimental methodologies are necessary to fully harness the power of these ...

My second installment of some interesting research papers that I have read over the past few weeks and would like to share with my community.

Copper is an essential trace element for normal development and function throughout the body, including the central nervous system (CNS). Alterations to cellular copper levels result in severe neurol...