scholarly journals Gene therapy for cystic fibrosis: new tools for precision medicine

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Jin-A Lee ◽  
Alex Cho ◽  
Elena N. Huang ◽  
Yiming Xu ◽  
Henry Quach ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Gene Therapy ◽  
Animal Models ◽  
Precision Medicine ◽  
Gene Targeting ◽  
New Models ◽  
Recent Developments ◽  
The Mean ◽  
Insight Into ◽  
The Way

AbstractThe discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible progress in treating the disease such that the mean survival age of individuals living with CF is now ~58 years in Canada. Recent developments in gene targeting tools and new cell and animal models have re-ignited the search for a permanent genetic cure for all CF. In this review, we highlight some of the more recent gene therapy approaches as well as new models that will provide insight into personalized therapies for CF.

scholarly journals Advanced Pediatric Diffuse Pontine Glioma Animal Models Pave the Way Towards Precision Medicine

2021 ◽  
Author(s):  
Zirong Chen ◽  
Peng Peng ◽  
Xiaolin Zhang ◽  
Barbara Mania-Farnell ◽  
Guifa Xi ◽  
...  
Keyword(s):  
Animal Models ◽  
Precision Medicine ◽  
Mouse Models ◽  
Pediatric Brain Tumors ◽  
Research Progress ◽  
Diffuse Pontine Glioma ◽  
Posttranslational Histone Modifications ◽  
Diffuse Intrinsic Pontine Gliomas ◽  
H3k27m Mutation ◽  
The Way

Diffuse intrinsic pontine gliomas (DIPGs) account for ~15% of pediatric brain tumors, which invariably present with poor survival regardless of treatment mode. Several seminal studies have revealed that 80% of DIPGs harbor H3K27M mutation coded by HIST1H3B, HIST1H3C and H3F3A genes. The H3K27M mutation has broad effects on gene expression and is considered a tumor driver. Determination of the effects of H3K27M on posttranslational histone modifications and gene regulations in DIPG is critical for identifying effective therapeutic targets. Advanced animal models play critical roles in translating these cutting-edge findings into clinical trial development. Here, we review current molecular research progress associated with DIPG. We also summarize DIPG animal models, highlighting novel genomic engineered mouse models (GEMMs) and innovative humanized DIPG mouse models. These models will pave the way towards personalized precision medicine for the treatment of DIPGs.

Hybrid nanotechnology and peptide nucleic acid could improve the effectiveness of gene therapy by increasing its cell permeability

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Carbon Nanotubes ◽  
Gene Therapy ◽  
Nucleic Acid ◽  
Peptide Nucleic Acid ◽  
Intracellular Delivery ◽  
Cell Permeability ◽  
Full Potential ◽  
Hybrid Nanotechnology ◽  
Insight Into ◽  
The Way

The utilization of nanoparticles and carbon nanotubes in PNA-based technologies has opened the way for the application of nanotechnology in oligonucleotide compilation. It is possible that further study may offer insight into how to improve the effectiveness of oligonucleotides by increasing their cell permeability. Because PNA accumulates within the cell when it is transported by a lipid-based carrier, it has insufficient cytosolic dispersion, which greatly limits its medicinal potential. This finding suggests that nanoparticles and nanostructures that are similar in appearance to carbon nanotubes can be employed to efficiently disseminate PNA. Innovative ways of intracellular delivery of PNA, such as the use of viruses and nutrients, will require significant and in-depth exploration before they can reach their full potential.

L’invenzione come forma di resistenza. Le attrezzature, i servizi e la produzione nelle Alpi

ARCHALP ◽  
2021 ◽  
Author(s):  
Keyword(s):  
Social Life ◽  
Economic Perspective ◽  
Productive Activities ◽  
New Models ◽  
Recent Developments ◽  
The Alps ◽  
Points Of View ◽  
New Generation ◽  
The Way

As Werner Bätzing (2003) points out in his book on Alpine geography, producers who do not emigrate and stay in the Alps do not act from an economic perspective but for social and cultural reasons. To resist the climatic, morphological and settlement difficulties, Alpine producers have always been driven to innovate, experimenting with new models, combining various forms of activity and independently creating the services necessary for their activity and social life. Today, after decades characterised by the abandonment of productive activities in the mountains, inventions as a form of resistance is once again one of the main themes of contemporary Alpine spaces. This chapter explores the issue of innovations in equipment, services and production in the Alps from two points of view. The first is that of a new generation of producers, entrepreneurs and project developers who are changing the way of producing in the mountains, by creating networks, sectors and services in the territory. The second is that of architects who experiment with new typological variations and constructive processes on the theme of production buildings in line with recent developments in ways of producing and working.

scholarly journals Advanced Pediatric Diffuse Pontine Glioma Murine Models Pave the Way towards Precision Medicine

Cancers ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 1114
Author(s):  
Zirong Chen ◽  
Peng Peng ◽  
Xiaolin Zhang ◽  
Barbara Mania-Farnell ◽  
Guifa Xi ◽  
...  
Keyword(s):  
Animal Models ◽  
Precision Medicine ◽  
Mouse Models ◽  
Pediatric Brain Tumors ◽  
Research Progress ◽  
Diffuse Pontine Glioma ◽  
Posttranslational Histone Modifications ◽  
Diffuse Intrinsic Pontine Gliomas ◽  
H3k27m Mutation ◽  
The Way

Diffuse intrinsic pontine gliomas (DIPGs) account for ~15% of pediatric brain tumors, which invariably present with poor survival regardless of treatment mode. Several seminal studies have revealed that 80% of DIPGs harbor H3K27M mutation coded by HIST1H3B, HIST1H3C and H3F3A genes. The H3K27M mutation has broad effects on gene expression and is considered a tumor driver. Determination of the effects of H3K27M on posttranslational histone modifications and gene regulations in DIPG is critical for identifying effective therapeutic targets. Advanced animal models play critical roles in translating these cutting-edge findings into clinical trial development. Here, we review current molecular research progress associated with DIPG. We also summarize DIPG animal models, highlighting novel genomic engineered mouse models (GEMMs) and innovative humanized DIPG mouse models. These models will pave the way towards personalized precision medicine for the treatment of DIPGs.

scholarly journals Pitch Change in Dog-Directed Speech

2015 ◽  
Vol 1 ◽  
pp. 28-35
Author(s):  
Christy C. Ringrose
Keyword(s):  
Social Norms ◽  
Fundamental Frequency ◽  
Speech Acts ◽  
High Pitch ◽  
Pitch Range ◽  
Pitch Change ◽  
The Mean ◽  
Speech Situation ◽  
Insight Into ◽  
The Way

Humans cannot help changing their speech in different situations. This kind of ‘intraspeaker variation’ happens every day when news-reporters turn from talking to their colleagues to talking to the camera, or when people suddenly start speaking in a strong dialect when talking with their family. People not only change the way they speak to different people, they also change the way they speak when not talking to people at all. This study examines one speaker and her dog, the goal being to find out why humans change the way they speak when talking to dogs. Dog-Directed Speech (DDS) is characterised by having a high fundamental frequency (pitch), as well as large amounts of questions and imperative utterances (Hirsh-Pasek and Treiman 1982, Burnham et al. 2002, Mitchell 2001). In order to take both of these features into account, we measured and compared the mean pitch of different speech acts which we expected to be common in DDS: disapproving utterances (“Stay”), approving utterances (“Good boy!”), and questions (“Who’s a good boy?”). We wanted to see whether the frequency of certain speech acts in DDS has anything to do with its high pitch and high pitch range. Although we did find variation in the pitch of different speech acts, we do not think that this explains the high pitch and pitch range of DDS. Rather, we conclude that DDS is motivated by social norms associated with talking to dogs. Our findings can give us insight into sociolinguistics in general because they show us that we respond to social norms that come with the speech situation, as well as just responding to the way our audience talks.

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