Shooting the Messenger: Locana Bio Writes a New Script for RNA Diseases

Based in San Diego, Locana Bio is built on the concept that gene therapy can deliver RNA-directed proteins to prevent or treat disease by targeting and modifying dysfunctional RNA.
Protein Structure

Base Editing Shows Promise for Progeria in Mouse Model

A new study provides further validation for the therapeutic potential of base editing, a technology that can specifically correct a common class of base substitutions without cleaving DNA. The work demonstrated for the first time the successful application of base editing in a mouse model of progeria, a premature aging disorder.
Medical technology concept. Gene therapy. Electronic medical record.

The Discovery Labs as a Cell and Gene Therapy Superstore

In addition to manufacturing, experts at the center focus on supporting analytical development and testing to help firms commercialize their cell and gene therapies.
OneMP Mass Photomete

“Weighing” for Viral Capsids Wins Best New Technology Award

A novel technique for measuring whether viral capsids are empty or full has won an award for best new bioprocessing technology.
DNA and medical technology concept, biotechnology, gene recombination

Optogenetics Turns Gut Bacterial Genes On and Off Inside Worms

Optogenetics can be used to achieve quantitative and temporal control of gut bacterial metabolism in order to reveal its local and systemic effects on host health and aging.
Toxoplasma gondii on colorful background

Toxoplasmosis Vaccine for Cats Benefits from Stealthy CRISPR-Cas9 Technique

A streamlined CRISPR-Cas9 approach may be used to develop a live vaccine against toxoplasmosis. This approach avoids unwanted genetic changes by eschewing plasmid vectors and the constitutive expression of Cas9. Instead, it takes advantage of electroporation and synthetic, chemically stabilized single-guide RNAs.

Building the Pipeline: Passage Bio Launches Manufacturing Operations

Gene therapy developers have been ringing out 2020 with a series of announcements about new manufacturing facilities, or at least production agreements with CDMOs. Thermo Fisher will build at its campus in Carlsbad, CA, while Taysha Gene Therapies disclosed plans in Durham, NC. Also, Passage Bio has announced two manufacturing developments at Catalent Cell & Gene Therapy’s Harmans/BWI campus in Harmans, MD and at the Princeton West Innovation Campus in Hopewell, NJ.

Three of a Kind: New Study Grades Methods to Assess Off-Target CRISPR Edits

Researchers at CRISPR Therapeutics compare three homology-independent off-target assessment methods– GUIDE-seq, CIRCLE-seq and SITE-seq–and infer all three methods are competent means of nominating off-target sites, validating CRISPR-Cas9 as a platform for the development of clinical therapeutics.
Adeno-associated virus, illustration

AAV Based Gene Therapy for Rare Genetic Disorders: Strategies and Challenges

In this GEN webinar, sponsored by WuXi AppTec, we will review the challenges of utilizing AAV, as well as discuss in detail the recent progress and outstanding questions in AAV-based gene therapy.

CiBER-seq Dissects Genetic Networks Using CRISPRi

A team at the University of California, Berkeley, has developed a method, called CiBER-seq, to rapidly determine the DNA sequences in the genome that regulate the expression of a specific gene. The technique is based on CRISPR genome editing, and links targeted, genome-wide genetic perturbations with a deep-sequencing readout.

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