Lifeboat Foundation

CRISPR has transformed gene editing, but still presents challenges in hard-to-transfect cells, such as pluripotent stem cells and primary cells.¹ The key to obtaining successful transfection in these cells lies in innovative workflows. Here Georges Müller, CEO and cofounder of SEED Biosciences, shares his perspective on why focusing on editing a single cell, rather than bulk cells, is a pivotal strategy to optimise CRISPR delivery.

Delivery of ribonucleoprotein (RNP) into cells is an essential factor for successful CRISPR gene editing. However, this is difficult to guarantee using traditional CRISPR gene editing methods, especially in hard-to-transfect cells. The standard CRISPR technique involves gathering a group of cells and then electroporating them, using short high-voltage pulses to overcome the barrier of their cell membranes. This allows bulk transfection of ribonucleoprotein (RNP) into the cells and then hopefully, nuclear translocation.

In this special episode, we’re joined by Cytosurge CSO Tobias Beyer, Ph.D., and SEED Biosciences CEO and Co-Founder Georges Muller, Ph.D., for an overview of gene editing with Cytosurge’s FluidFM® in combination with DispenCell™ dispensing technologies.

Tobias and Georges explain the FluidFM® technique and how it differs from traditional CRISPR methods along with the advantages the technology has over other methods of gene editing.

Continue reading “The Ins and Outs of Single Cell Gene Editing (Sponsored by Molecular Devices)” »

Rice University engineers have developed the smallest implantable brain stimulator demonstrated in a human patient. Thanks to pioneering magnetoelectric power transfer technology, the pea-sized device developed in the Rice lab of Jacob Robinson in collaboration with Motif Neurotech and clinicians Dr. Sameer Sheth and Dr. Sunil Sheth can be powered wirelessly via an external transmitter and used to stimulate the brain through the dura ⎯ the protective membrane attached to the bottom of the skull.

The device, known as the Digitally programmable Over-brain Therapeutic (DOT), could revolutionize treatment for drug-resistant depression and other psychiatric or neurological disorders by providing a therapeutic alternative that offers greater patient autonomy and accessibility than current neurostimulation-based therapies and is less invasive than other brain-computer interfaces (BCIs).

Continue reading “Rice team demonstrates miniature brain stimulator in humans” »

A first-ever dataset bridging molecular information about the poplar tree microbiome to ecosystem-level processes has been released by a team of Department of Energy scientists led by Oak Ridge National Laboratory. The project aims to inform research regarding how natural systems function, their vulnerability to a changing climate, and ultimately how plants might be engineered for better performance as sources of bioenergy and natural carbon storage.

The data, described in Nature Publishing Group’s Scientific Data, provides in-depth information on 27 genetically distinct variants, or genotypes, of Populus trichocarpa, a poplar tree of interest as a bioenergy crop. The genotypes are among those that the ORNL-led Center for Bioenergy Innovation previously included in a genome-wide association study linking genetic variations to the trees’ physical traits. ORNL researchers collected leaf, soil and root samples from poplar fields in two regions of Oregon — one in a wetter area subject to flooding and the other drier and susceptible to drought.

Details in the newly integrated dataset range from the trees’ genetic makeup and gene expression to the chemistry of the soil environment, analysis of the microbes that live on and around the trees and compounds the plants and microbes produce.

Gene therapy may be the best hope for curing retinitis pigmentosa (RP), an inherited condition that usually leads to severe vision loss and blinds 1.5 million people worldwide.

But there’s a huge obstacle: RP can be caused by mutations in over 80 different genes. To treat most RP patients with gene therapy, researchers would have to create a therapy for each gene—a nearly impractical task using current gene therapy strategies.

A more universal treatment may be forthcoming. Using CRISPR-based genome engineering, scientists at Columbia University Vagelos College of Physicians and Surgeons are designing a gene therapy with the potential to treat RP patients regardless of the underlying genetic defect.

Using this natural process as a basis, scientists developed a gene-editing tool called CRISPR/Cas that can cut a specific DNA sequence by simply providing it with an RNA template of the target sequence. This allows scientists to add, delete, or replace elements within the target DNA sequence. Slicing a specific part of a gene’s DNA sequence with the help of the Cas9 enzyme, aids in DNA repair.

This system represented a big leap from previous gene-editing technologies, which required designing and making a custom DNA-cutting enzyme for each target sequence rather than simply providing an RNA guide, which is much simpler to synthesize.

CRISPR gene editing has already changed the way scientists do research, allowing a wide range of applications across multiple fields. Here are some of the diseases that scientists aim to tackle using CRISPR/Cas technology, testing its possibilities and limits as a medical tool.

While during Apollo 13 the phrase “Failure is Not an Option” was coined, in life and especially for students, failure must be an option for growth. In this talk, Michelle Lucas encourages failing forward. Michelle Lucas was raised in the Chicagoland area and found a passion for space very early in her life. She studied Aerospace Engineering, Communications & Space Studies at Purdue University and Embry Riddle Aeronautical University. During this time she conducted microgravity fluids research on NASA’s KC-135 aircraft and also worked as a counselor at Space Camp in Florida. After graduation from college, Michelle spent 11 years working at NASA’s Johnson Space Center. She began on the Safety Reliability & Quality Assurance Contract as part of the Payload Safety Review Panel for experiments flying to the International Space Station. After this she worked as a Flight Controller in Mission Control for the International Space Station for the Ops Plan Group and as a Astronaut Technical Instructor in the Daily Operations Group. Additionally she worked with each of the International Partners (European Space Agency – ESA, Japanese Space Agency – JAXA and the Russian Space Agency) in the field of Daily Operations, Flight Controller and Instructor Training. Michelle was responsible for the basic instructional training of all technical instructors for in the US as well as for the ISS International Partners. Michelle was part of the Core NASA Extreme Environment Mission Operations (NEEMO) team for 9 missions where astronauts would carry out analog space missions underwater in the Aquarius habitat. Along the way, Michelle found she has a passion for exciting the next generation and founded the non-profit Higher Orbits to use space to excite and inspire students about STEM, STEAM, Leadership, Teambuilding and Communication. Higher Orbits flagship program is called Go For Launch! This program allows students work with an astronaut and other accomplished individuals in the fields of Space, STEM and STEAM. Additionally, Michelle and a business partner run uniphi space agency – a talent management company for retired astronauts. Michelle is proud to be a Space Camp Alumni and member of the Space Camp Hall of Fame and believes that collaboration in space and STEM is the key to the stars! Space Inspires! This talk was given at a TEDx event using the TED conference format but independently organized by a local community.

Wires and cables are not the only things that can get entangled: Plants, fungi, and bacteria can all exhibit filamentous or branching growth patterns that eventually become entangled too. Previous work with nonliving materials demonstrated that entanglement can produce unique and desirable material properties, but achieving entanglement requires meticulously engineered material structure and geometry. It has been unclear if the same rules apply to organisms, which, unlike nonliving systems, develop through a process of progressive growth. Through a blend of experiments and simulations, we show that growth easily produces entanglement.

Specifically, we find that treelike growth leads to branch arrangements that cannot be disassembled without breaking or deforming branches. Further, entanglement via growth is possible for a wide range of geometries. In fact, it appears to be largely insensitive to the geometry of branched trees but instead depends sensitively on how long the organism can keep growing. In other words, growing branched trees can entangle with almost any geometry if they keep growing for a long-enough time.

Entanglement via growth appears to be largely distinct from, and easier to achieve than, entanglement of nonliving materials. These observations may in part account for the broad prevalence of entanglement in biological systems, as well as inform recent experiments that observed the rapid evolution of entanglement, though much still remains to be discovered.

PHILADELPHIA — Scientists at the University of Pennsylvania’s Perelman School of Medicine have developed a new method to create human artificial chromosomes (HACs) that could revolutionize gene therapy and other biotechnology applications. The study, published in Science, describes an approach that efficiently forms single-copy HACs, bypassing a common hurdle that has hindered progress in this field for decades.

Artificial chromosomes are lab-made structures designed to mimic the function of natural chromosomes, the packaged bundles of DNA found in the cells of humans and other organisms. These synthetic constructs have the potential to serve as vehicles for delivering therapeutic genes or as tools for studying chromosome biology. However, previous attempts to create HACs have been plagued by a major issue: the DNA segments used to build them often link together in unpredictable ways, forming long, tangled chains with rearranged sequences.

The Penn Medicine team, led by Dr. Ben Black, sought to overcome this challenge by completely overhauling the approach to HAC design and delivery. “The HAC we built is very attractive for eventual deployment in biotechnology applications, for instance, where large-scale genetic engineering of cells is desired,” Dr. Black explains in a media release. “A bonus is that they exist alongside natural chromosomes without having to alter the natural chromosomes in the cell.”