Christopher E. Nelson
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Beyond the Cut: Long-read sequencing reveals complex genomic and transcriptomic changes in AAV-CRISPR therapy for Duchenne Muscular Dystrophy Open
Adeno associated virus (AAV)-mediated delivery of CRISPR associated nucleases (AAV-CRISPR) is a promising solution to treat genetic diseases such as Duchenne Muscular Dystrophy (DMD) and is now in early clinical trials. However, genotoxici…
Standardizing a Protocol for Streamlined Synthesis and Characterization of Lipid Nanoparticles to Enable Preclinical Research and Education Open
Lipid nanoparticles (LNPs) have revolutionized nucleic acid delivery, enabled the first FDA-approved RNAi therapy (Onpattro), and accelerated the development of mRNA vaccines during the COVID-19 pandemic. The success of LNP-based vaccines …
A dual-fluorescence assay for gene delivery vehicle screening in macrophages with an inflammation-inducible reporter construct Open
The online version contains supplementary material available at 10.1186/s44330-025-00030-x.
Preclinical development of genome editing to treat Duchenne muscular dystrophy by exon skipping Open
Duchenne muscular dystrophy (DMD) is caused by loss-of-function mutations to the gene encoding dystrophin. Restoring the reading frame of dystrophin by removing internal out-of-frame exons may address symptoms of DMD. Therefore, the princi…
Resistance to Acetyl Coenzyme A Carboxylase (ACCase) Inhibitor in Lolium multiflorum: Effect of Multiple Target-Site Mutations Open
Italian ryegrass (Lolium multiflorum Lam.) is a persistent weed species that poses significant management challenges in key agricultural crops such as wheat, corn, cotton, and soybean. This study investigated the prevalence of resistance t…
Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci Open
Gene replacement therapies primarily rely on adeno-associated virus (AAV) vectors for transgene expression. However, episomal expression can decline over time due to vector loss or epigenetic silencing. CRISPR-based integration methods off…
A Dual-Fluorescence Assay for Gene Delivery Vehicle Screening in Macrophages with an Inflammation-Inducible Reporter Construct Open
Background Macrophages are a promising target for therapeutics in various applications such as regenerative medicine and immunotherapy for cancer. Due to their plastic nature, macrophages can switch from a non-activated state to activated …
Optimizing Recombinant Cas9 Expression: Insights from E. coli BL21(DE3) Strains for Enhanced Protein Purification and Genome Editing Open
The CRISPR-Cas9 system is a revolutionary tool in genetic engineering, offering unprecedented precision and efficiency in genome editing. Cas9, an enzyme derived from bacteria, is guided by RNA to edit DNA sequences within cells precisely.…
Precision and efficacy of RNA-guided DNA integration in high-expressing muscle loci Open
Gene replacement therapies in genetic medicine primarily rely on adeno-associated viral (AAV) vectors for transgene expression. However, episomal expression can decline over time due to epigenetic silencing. CRISPR-based integration method…
Delivery challenges for CRISPR—Cas9 genome editing for Duchenne muscular dystrophy Open
Duchene muscular dystrophy (DMD) is an X-linked neuromuscular disorder that affects about one in every 5000 live male births. DMD is caused by mutations in the gene that codes for dystrophin, which is required for muscle membrane stabiliza…
Longitudinal examination of perfusion and angiogenesis markers in primary colorectal tumors shows distinct signatures for metronomic and maximum-tolerated dose strategies Open
1 Abstract Metronomic chemotherapy (MET) has been developed to address the shortcomings of maximum-tolerated chemotherapy (MTD) in regard to toxicity and development of resistance mechanisms in the tumor. In colorectal cancer (CRC), MET is…
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy Open
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from …
View article: AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice Open
Adeno-associated virus-mediated (AAV-mediated) CRISPR editing is a revolutionary approach for treating inherited diseases. Sustained, often life-long mutation correction is required for treating these diseases. Unfortunately, this has neve…
Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma Open
Genetically engineered mouse models that employ site-specific recombinase technology are important tools for cancer research but can be costly and time-consuming. The CRISPR-Cas9 system has been adapted to generate autochthonous tumours in…
Local Delivery of PHD2 siRNA from ROS‐Degradable Scaffolds to Promote Diabetic Wound Healing Open
Small interfering RNA (siRNA) delivered from reactive oxygen species-degradable tissue engineering scaffolds promotes diabetic wound healing in rats. Porous poly(thioketal-urethane) scaffolds implanted in diabetic wounds locally deliver si…
Engineering Delivery Vehicles for Genome Editing Open
The field of genome engineering has created new possibilities for gene therapy, including improved animal models of disease, engineered cell therapies, and in vivo gene repair. The most significant challenge for the clinical translation of…
482. Local and Systemic Gene Editing in a Mouse Model of Duchenne Muscular Dystrophy Open
Duchenne muscular dystrophy (DMD) is a highly prevalent genetic disorder leading to muscle wasting, loss of ambulation, and premature death by the third decade of life. DMD is caused by gene deletions, duplications, or nonsense mutations l…
504. Restoration of Dystrophin Expression by Gene Editing with S. aureus Cas9 in Models of Duchenne Muscular Dystrophy Open
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease characterized by progressive muscle wasting, loss of ambulation, and typically death in the third decade of life due to respiratory and cardiac complications. DMD r…
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy Open
Duchenne muscular dystrophy (DMD) is a devastating disease affecting about 1 out of 5000 male births and caused by mutations in the dystrophin gene. Genome editing has the potential to restore expression of a modified dystrophin gene from …