Neurodegenerative disorders selectively affect vulnerable neurons, cause progressive loss of cognitive and/or motor functions, and pose significant healthcare challenges for societies with aging populations. Many of these diseases share clinical manifestations. For instance, cognitive deficits occur in Alzheimer’s diseases (AD) and frontotemporal dementia (FTD), while motor deficits occur in Parkinson’s disease (PD), amyotrophic lateral sclerosis (ALS), Huntington’s disease (HD), and spinocerebellar ataxias (SCAs). Decades of fundamental research have shifted towards the view that in addition to aggregation of mutant proteins, many cellular pathways are now known to be affected in neurodegeneration. The new insights into pathogenic contributors, such as chronic DNA damage activation, abnormal phase transition, defects in energy metabolism, and neuroinflammation, not only highlight the complexity of the diseases, but also identify novel therapeutic targets and commonly affected pathways that can help improve treatment efficacy and specificity.
This Research Topic aims to report recent discoveries of pathomechanisms and therapeutic innovations with an emphasis on polyglutamine (polyQ) diseases, AD and PD. Original research articles, review articles, and brief communications will be considered for publication.
This Research Topic will cover the following aspects:
- Modulation of DNA damage repair, protein quality control (ubiquitin-proteasome system and autophagy-lysosome pathway), mitochondrial homeostasis, liquid-liquid phase separation, stress granules, RNA binding proteins, RNA foci, immune activation, and repeat associated non-AUG (RAN) translation.
- Disease models ranging from cultured cells, 3D organoids, Caenorhabditis elegans, flies, to mice.
- Therapeutic strategies including CRISPR-Cas9 genome editing, Cas13 RNA editing, base editors, prime editors, CRISPR-associated transposons, small interfering RNA (siRNA), antisense oligonucleotides (ASOs), DNAzymes, ribozymes, aptamers, and small molecules.
- Computational or experimental identification of mutations that can facilitate allele-specific targeting.
- Therapeutic delivery systems including viral vectors and non-viral particles.
- Safety evaluation of therapeutic strategies.
Omics studies accepted in the 'Neurogenomics' section include proteomics - such as quantitative protein changes with pathway analysis, and novel protein interaction partners that contribute to disease mechanism or that serve as new drug targets, transcriptomics - such as changes in RNA expression (e.g., RNA isoform, lnRNA and miRNA) and alterations in transcription factor occupancy), and epigenomics (such as chromosome remodeling and RNA modification (e.g., m6A)).
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
Neurodegenerative disorders selectively affect vulnerable neurons, cause progressive loss of cognitive and/or motor functions, and pose significant healthcare challenges for societies with aging populations. Many of these diseases share clinical manifestations. For instance, cognitive deficits occur in Alzheimer’s diseases (AD) and frontotemporal dementia (FTD), while motor deficits occur in Parkinson’s disease (PD), amyotrophic lateral sclerosis (ALS), Huntington’s disease (HD), and spinocerebellar ataxias (SCAs). Decades of fundamental research have shifted towards the view that in addition to aggregation of mutant proteins, many cellular pathways are now known to be affected in neurodegeneration. The new insights into pathogenic contributors, such as chronic DNA damage activation, abnormal phase transition, defects in energy metabolism, and neuroinflammation, not only highlight the complexity of the diseases, but also identify novel therapeutic targets and commonly affected pathways that can help improve treatment efficacy and specificity.
This Research Topic aims to report recent discoveries of pathomechanisms and therapeutic innovations with an emphasis on polyglutamine (polyQ) diseases, AD and PD. Original research articles, review articles, and brief communications will be considered for publication.
This Research Topic will cover the following aspects:
- Modulation of DNA damage repair, protein quality control (ubiquitin-proteasome system and autophagy-lysosome pathway), mitochondrial homeostasis, liquid-liquid phase separation, stress granules, RNA binding proteins, RNA foci, immune activation, and repeat associated non-AUG (RAN) translation.
- Disease models ranging from cultured cells, 3D organoids, Caenorhabditis elegans, flies, to mice.
- Therapeutic strategies including CRISPR-Cas9 genome editing, Cas13 RNA editing, base editors, prime editors, CRISPR-associated transposons, small interfering RNA (siRNA), antisense oligonucleotides (ASOs), DNAzymes, ribozymes, aptamers, and small molecules.
- Computational or experimental identification of mutations that can facilitate allele-specific targeting.
- Therapeutic delivery systems including viral vectors and non-viral particles.
- Safety evaluation of therapeutic strategies.
Omics studies accepted in the 'Neurogenomics' section include proteomics - such as quantitative protein changes with pathway analysis, and novel protein interaction partners that contribute to disease mechanism or that serve as new drug targets, transcriptomics - such as changes in RNA expression (e.g., RNA isoform, lnRNA and miRNA) and alterations in transcription factor occupancy), and epigenomics (such as chromosome remodeling and RNA modification (e.g., m6A)).
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.
