Results 41 to 50 of about 33,232 (163)
A Database for TSSs of Human MicroRNAs [PDF]
, 2012 MicroRNAs (miRNAs) are small endogeneous non-coding RNAs of about 22nt length. These short RNAs regulate the expression of mRNAs by hybridizing with their 3'-UTRs or by translational repression.
Malay Bhattacharyya +1 more
core +2 more sources
Therapy for Myhre Syndrome: Goals, Misconceptions, and Current Agents
American Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.ABSTRACT Myhre Syndrome (MYHRS, MIM #139210) is a rare, multisystem connective tissue disorder caused by recurrent heterozygous gain‐of‐function pathogenic variants in the SMAD4 gene, a key player in TGF‐β signaling and a regulator of extracellular matrix homeostasis.
Alessandro De Falco +2 more
wiley +1 more source
Bacteria herald a new era of gene editing. [PDF]
, 2013 The demonstration that nucleases guided by bacterial RNA can disrupt human genes represents a landmark in the rapidly developing field of genome ...
Segal, David J
core
A small RNA response at DNA ends in Drosophila [PDF]
, 2012 Small RNAs have been implicated in numerous cellular processes, including effects on chromatin structure and the repression of transposons. We describe the generation of a small RNA response at DNA ends in Drosophila that is analogous to the recently ...
Böttcher, Romy +2 more
core +1 more source
Advanced NanoBiomed Research, EarlyView.CRISPR/Cas9 has revolutionized the field of gene therapy, but delivery remains an outstanding issue. We propose a nonviral gold‐nanoparticle platform for co‐delivery of CRISPR/Cas9 ribonucleoprotein and long 2.1 kilobase dsDNA transgene constructs. This CRISPR‐AuNP is inexpensive to produce and mediate gene editing and DNA delivery in T cells and CD34+
Rachel A. Cunningham +8 more
wiley +1 more source
Adaptation of intronic homing endonuclease for successful horizontal transmission [PDF]
, 2005 oai:kutarr.kochi-tech.ac.jp:00000010journal ...
10 +13 more
core +1 more source
A Mechanistic Model of rAAV Production in Synthetic Cell Lines
Biotechnology and Bioengineering, EarlyView.ABSTRACT The recombinant adeno‐associated virus (rAAV) is a widely used vector for gene therapy. Its manufacturing faces significant challenges in producing the large quantities of vectors needed for clinical applications and reducing empty particles.
Prahalad Srinivasan +5 more
wiley +1 more source
Genome Editing with Crispr-Cas9 Systems: Basic Research and Clinical Applications [PDF]
, 2017 BACKGROUND: Recently established genome editing technologies will open new avenues for biological research and development. Human genome editing is a powerful tool which offers great scientific and therapeutic potential.CONTENT: Genome editing using the ...
Dewi, N. M. (Nurrani) +2 more
core +4 more sources
Environmental Toxicology and Chemistry, EarlyView.Cross‐species extrapolation of adverse outcome pathway network on reproductive toxicity under the One Health perspective using new approach methodologies. AOP = adverse outcome pathway. Abstract Although ecotoxicological and toxicological risk assessments are performed separately from each other, recent efforts have been made in both disciplines to ...
Elizabeth Dufourcq Sekatcheff +2 more
wiley +1 more source
SSB-1 of the yeast Saccharomyces cerevisiae is a nucleolar-specific, silver-binding protein that is associated with the snR10 and snR11 small nuclear RNAs [PDF]
, 1990 SSB-1, the yeast single-strand RNA-binding protein, is demonstrated to be a yeast nucleolar-specific, silver-binding protein. In double-label immunofluorescence microscopy experiments antibodies to two other nucleolar proteins, RNA Pol I 190-kD and ...
Abelson, John +3 more
core +2 more sources