Chengzu Long
YOU?
Author Swipe
View article: Preventing large deletions and chromosome loss in engineered human primary T cells by CasPlus with optimized guide RNAs
Preventing large deletions and chromosome loss in engineered human primary T cells by CasPlus with optimized guide RNAs Open
Genetically engineered T-cell therapies rely heavily on genome editing tools, such as the CRISPR/Cas9 system. However, unintended on-target chromosomal alterations, including large deletions and chromosome loss can occur and pose significa…
View article: Modeling and correction of protein conformational disease in iPSC-derived neurons through personalized base editing
Modeling and correction of protein conformational disease in iPSC-derived neurons through personalized base editing Open
Altered protein conformation can cause incurable neurodegenerative disorders. Mutations in SERPINI1, the gene encoding neuroserpin, can alter protein conformation resulting in cytotoxic aggregation leading to neuronal death. Familial encep…
View article: T4 DNA polymerase prevents deleterious on-target DNA damage and enhances precise CRISPR editing
T4 DNA polymerase prevents deleterious on-target DNA damage and enhances precise CRISPR editing Open
Unintended on-target chromosomal alterations induced by CRISPR/Cas9 in mammalian cells are common, particularly large deletions and chromosomal translocations, and present a safety challenge for genome editing. Thus, there is still an unme…
View article: Modeling and Correction of Protein Conformational Disease in iPSC-derived Neurons through Personalized Base Editing
Modeling and Correction of Protein Conformational Disease in iPSC-derived Neurons through Personalized Base Editing Open
Altered protein conformation can cause incurable neurodegenerative disorders. Mutations in SERPINI1 , the gene encoding neuroserpin, can alter protein conformation resulting in cytotoxic aggregation leading to neuronal death. Familial ence…
View article: Phage DNA polymerase prevents deleterious on-target DNA damage and enhances precise CRISPR/Cas9 editing
Phage DNA polymerase prevents deleterious on-target DNA damage and enhances precise CRISPR/Cas9 editing Open
Common unintended chromosomal alterations induced by CRISPR/Cas9 in mammalian cells, particularly on-target large deletions and chromosomal translocations present a safety challenge for genome editing. Base editing and prime editing that c…
View article: <i>PINK1</i> gene mutation by pair truncated sgRNA/Cas9-D10A in cynomolgus monkeys
<i>PINK1</i> gene mutation by pair truncated sgRNA/Cas9-D10A in cynomolgus monkeys Open
Mutations of PTEN-induced kinase I (PINK1) cause early-onset Parkinson's disease (PD) with selective neurodegeneration in humans. However, current PINK1 knockout mouse and pig models are unable to recapitulate the typical neurodegenerative…
View article: Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases
Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases Open
There are an estimated 10 000 monogenic diseases affecting tens of millions of individuals worldwide. The application of CRISPR/Cas genome editing tools to treat monogenic diseases is an emerging strategy with the potential to generate per…
View article: Generation of a Hutchinson–Gilford progeria syndrome monkey model by base editing
Generation of a Hutchinson–Gilford progeria syndrome monkey model by base editing Open
Many human genetic diseases, including Hutchinson-Gilford progeria syndrome (HGPS), are caused by single point mutations. HGPS is a rare disorder that causes premature aging and is usually caused by a de novo point mutation in the LMNA gen…
View article: Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing
Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing Open
Muscular dystrophies represent a large group of genetic disorders that significantly impair quality of life and often progress to premature death. There is no effective treatment for these debilitating diseases. Most therapies, developed t…
View article: Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing
Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing Open
CRISPR-mediated genome editing efficiently corrected cardiac abnormalities associated with DMD in human engineered heart tissue.
View article: Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy Open
Single-cut correction of a dystrophin gene mutation with CRISPR/Cas9 restored dystrophin expression in skeletal and cardiac muscles in a mouse model of Duchenne muscular dystrophy.
View article: Functional correction of dystrophin actin binding domain mutations by genome editing
Functional correction of dystrophin actin binding domain mutations by genome editing Open
Dystrophin maintains the integrity of striated muscles by linking the actin cytoskeleton with the cell membrane. Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene (DMD) that result in progressive, debilitating…
View article: CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice Open
CRISPR-Cpf1–mediated correction of Duchenne muscular dystrophy mutations in human cells and a mouse model.
View article: Genome Editing of Monogenic Neuromuscular Diseases
Genome Editing of Monogenic Neuromuscular Diseases Open
Genome editing is a rapidly evolving technology with enormous translational potential once efficacy, delivery, and safety issues are addressed. The clinical impact of this technology is that genome editing can permanently correct disease-c…
View article: Effects of dietary pyrroloquinoline quinone disodium on growth, carcass characteristics, redox status, and mitochondria metabolism in broilers
Effects of dietary pyrroloquinoline quinone disodium on growth, carcass characteristics, redox status, and mitochondria metabolism in broilers Open
The potential benefits of supplementing pyrroloquinoline quinone disodium (PQQ·Na2) in the diet of broiler chicks were explored. We first examined the effect of different levels of dietary PQQ·Na2 on growth performance, carcass characteris…