Results 111 to 120 of about 267,421 (305)

Suppression of HBV replication by the expression of nickase- and nuclease dead-Cas9

Scientific Reports, 2017
Complete removal of hepatitis B virus (HBV) DNA from nuclei is difficult by the current therapies. Recent reports have shown that a novel genome-editing tool using Cas9 with a single-guide RNA (sgRNA) system can cleave the HBV genome in vitro and in vivo.
Takeshi Kurihara, Takasuke Fukuhara, Chikako Ono, Satomi Yamamoto, Kentaro Uemura, Toru Okamoto, Masaya Sugiyama, Daisuke Motooka, Shota Nakamura, Masato Ikawa, Masashi Mizokami, Yoshihiko Maehara, Yoshiharu Matsuura   +12 more
doaj   +1 more source

Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9 [PDF]

, 2018
Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons.
Cappellari, O, Cho, H-Y, Dickson, G, Kim, D, Kim, E, Kim, J-S, Koo, T, Lu-Nguyen, N B, Malerba, A, Popplewell, L   +9 more
core   +2 more sources

Developmental and Epileptic Encephalopathy due to Biallelic Pathogenic Variants in PIGM

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective PIGM encodes a critical enzyme in the glycosylphosphatidylinositol (GPI)‐anchor biosynthesis pathway. While promoter‐region mutations in PIGM have been associated with a relatively mild phenotype characterized by portal vein thrombosis and absence seizures, recent evidence suggests that coding‐region mutations result in a more severe
Júlia Sala‐Coromina, Anna Marcé‐Grau, Barbara Masotto, Marta Codina, Lamia BenJemaa, Yasmina Elaribi, Mónica Martinez‐Gallo, Roger Colobran, Angel Sanchez‐Montañez, Irene Valenzuela, Yoshiko Murakami, Alfons Macaya   +11 more
wiley   +1 more source

Embryos as Patients? Medical Provider Duties in the Age of CRISPR/Cas9 [PDF]

, 2017
The CRISPR/Cas9 genome engineering platform is the first method of gene editing that could potentially be used to treat genetic disorders in human embryos. No past therapies, genetic or otherwise, have been intended or used to treat disorders in existent
Powell, G. Edward, III
core   +1 more source

Integrate CRISPR/Cas9 for protein expression of HLA-B*38:68Q via precise gene editing. [PDF]

, 2019
The determination of null- or low-expressed HLA alleles is clinically relevant in both hematopoietic stem cell transplantation and solid organ transplantation.
Reed, Elaine F, Yin, Yuxin, Zhang, Qiuheng   +2 more
core   +1 more source

Cardiac‐Derived ECM Microspheres for Enhanced hiPSC‐CMs Maturation

Advanced Functional Materials, EarlyView.
Cardiac extracellular matrix microspheres derived from decellularized porcine heart provide a biomimetic 3D microenvironment for human induced pluripotent stem cell–derived cardiomyocytes (hiPSC‐CMs). This platform supports short‐ and long‐term culture, enhances structural organization, and promotes electrophysiological and functional maturation of ...
Jiazhu Xu, Joel Aboagye, Marcella Edwards, Nick Rogozinski, Yufeng Wen, Angello Huerta Gomez, Zui Pan, Ge Zhang, Huaxiao Yang, Yi Hong   +9 more
wiley   +1 more source

VIVID: A qPCR‐Based Platform for Sensitive and Quantitative In Vivo Tracking of Extracellular Vesicles

Advanced Functional Materials, EarlyView.
This study introduces VIVID (Vesicle In Vivo Identification using DNA), a qPCR‐based platform that tracks PCR‐amplifiable DNA tags loaded in the EVs for accurate and quantifiable EV biodistribution in vivo. ABSTRACT Extracellular vesicles (EVs) represent promising carriers for nucleic acid therapeutics, offering advantages over synthetic nanoparticles ...
Oscar Boyadjian, Talin Ebrahimian, Jonathan Miranda O'Connor, Janusz Rak, Stephanie Lehoux, Maryam Tabrizian   +5 more
wiley   +1 more source

Structure Formation in Butterfly Scales: Interplay of Genetic Control, Mechanical Instabilities, and Dynamic Material Properties

Advanced Functional Materials, EarlyView.
Butterfly wing scales are intricate cuticular functional nanosctructures. This perspective suggests that spatially varying material properties, cytoskeletal constraints, and growth‐driven mechanical instabilities shape the resulting nanoscale architectures created from single cells.
Anupama Prakash, Victoria Lloyd, Yi Yang, Anna‐Lee Jessop, Anthony McDougal, Primož Pirih, Guillaume Duclos, Andrew Parnell, Mathias Kolle, Nicola J. Nadeau, Bodo D. Wilts   +10 more
wiley   +1 more source

CRISPR-Cas revolution in Agriculture: From precision genome editing to sustainable crop improvement

Current Innovation in Agriculture Science
The CRISPR-Cas system has emerged as a transformative tool in agricultural biotechnology, revolutionizing the landscape of crop improvement. This review paper explores the multifaceted applications of CRISPR technology in agriculture, from its ...
Anantha Rama A
doaj  

The aromatic amino acid hydroxylase genes AAH1 and AAH2 in Toxoplasma gondii contribute to transmission in the cat [PDF]

, 2017
The Toxoplasma gondii genome contains two aromatic amino acid hydroxylase genes, AAH1 and AAH2 encode proteins that produce L-DOPA, which can serve as a precursor of catecholamine neurotransmitters.
Dubey, Jitender P, Sibley, L. David, Verma, Shiv K, Wang, Zi T   +3 more
core   +6 more sources