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Whole-genome phenotype prediction with machine learning: open problems in bacterial genomics.

MOTIVATION: How can we identify causal genetic mechanisms governing bacterial traits? Initial efforts entrusting machine learning models to handle the task of predicting phenotype from genotype yield high accuracy scores. However, attempts to extract meaningful interpretations from the predictive models are found to be corrupted by falsely identified 'causal' features. Relying solely on pattern recognition and correlations is unreliable, significantly so in bacterial genomics settings where high-dimensionality and spurious associations are the norm. Though it is not yet clear whether we can overcome this hurdle, significant efforts are being made towards discovering potential high-risk bacterial genetic variants. In view of this, we set up open problems surrounding phenotype prediction from bacterial whole-genome datasets and extending those approaches to learning causal effects, and discuss challenges that impact the reliability of a machine's decision-making when faced with datasets of this nature. RESULTS: We identify major sources of non-injectivity in the formulation of the genotype-to-phenotype mapping function-linkage-disequilibrium, limited sampling, information loss in representations, unmeasured confounders and observational noise-and analyse their implications for machine learning applications. Using a collection of 4,140 Staphylococcus aureus isolates, we illustrate challenges surrounding the defined open problems. AVAILABILITY AND IMPLEMENTATION: Raw sequencing data are available from the European Nucleotide Archive (ENA) under project accessions ERP001012, PRJEB3174, PRJEB2655, PRJEB2756, and PRJEB2944. Assemblies and annotations were generated with the Sanger bacterial pipeline (https://github.com/sanger-pathogens/vr-codebase) and unitigs extracted using DBGWAS (https://gitlab.com/leoisl/dbgwas).

Machine Learning↗

EAE: a model for immune intervention with synthetic peptides.

The cellular and molecular requirements for the autoimmune disease EAE are being defined in increasing detail through intense scrutiny of critical autoantigenic peptides, class II MHC molecules, and alpha beta TCRs involved in the disease process. This study has led to novel immunotherapeutic approaches, many of which are based on the administration of synthetic peptides. Since short peptides are understood to be the minimal antigenic units bound by MHC molecules for recognition by T cells, they are attractive experimental tools for finely modulating specific immune responses. It is clear that a large number of defined peptides can dramatically influence the course of EAE. Table IV lists a number of potential mechanisms which may mediate disease prevention. Increasing evidence supports the idea that prevention of autoimmune disease can result from MHC-blockade by peptides which competitively bind to class II molecules. However, for some peptides such as the perplexing partial agonist Ac1-11[4A], the mechanism by which these precisely defined units act is not yet fully understood. Numerous hurdles hinder immediate clinical application of peptide-based immunotherapy. Nevertheless, the knowledge gained by probing experimental autoimmunity with defined peptides promises to inspire original and practical approaches to treating human autoimmune disease.

Amino Acid Sequence↗

Vaccine research and development for the prevention of filarial nematode infections.

The development of vaccines for the prevention of filarial nematode infections is in a state of relative infancy in comparison to vaccines for other parasitic diseases, such as schistosomiasis and malaria. There are many reasons for this slow start. Some of the principal problems are: (1) the lengthy and complex life cycle of these organisms with attendant complex immune responses, (2) the unique characteristics associated with a relatively large number of different pathogens, (3) the lack of suitable model systems for study of medically important infections, (4) the paucity of parasite material for antigen discovery and recombinant library construction, (5) the lack of substantial evidence suggesting the natural occurrence of protective immune responses, and (6) the limited data on mechanisms responsible for protective immunity. As technical hurdles are considered, it is also critical to focus on the characteristics of a vaccine necessary for its eventual utility. In the case of a vaccine for D. immitis a completely successful product will need to approach a 99+% efficacy. This is because of the 99+% efficacy of competitive chemotherapeutic products and the fact that microfilaremia observed on blood examination, resulting from as few as two worms, would present as a vaccine failure. Although very low worm burdens in large dogs could be perceived as success in the context of protection from clinical disease, because of the option of virtually complete chemoprophylactic protection, the typical veterinary practitioner would probably fail to appreciate less than complete vaccine protection. In contrast, a vaccine that produced a reduction in adult worm burdens without complete protection in either lymphatic filariasis or onchocerciasis would be very important. Highly effective chemoprophylactic agents are not widely available for prevention of the human filariases, and dramatically reduced clinical disease provided by less than a completely effective vaccine could occur as the result of fewer adult worms. The importance of developing these vaccines has outweighed the obstacles to this research. There has been a great deal of epidemiological and experimental evidence to suggest a vaccine is feasible and antigen discovery has progressed relatively rapidly within just the past few years. Efforts to generate appropriate larval cDNA libraries are beginning to yield dividends and a variety of fascinating vaccine candidates have been cloned. Additional antigen discovery, research on appropriate modalities for overexpression of genes from these parasites, and the complex tasks associated with vaccinology remain as significant research and development obstacles.(ABSTRACT TRUNCATED AT 400 WORDS)

Animals↗

Development and validation of a novel LC-MS/MS method for simultaneous quantification of fidaxomicin and metabolite (OP-1118) from feces for gut pharmacobiome studies.

Fidaxomicin is a first-line antibiotic for treating Clostridioides difficile infection. While it has low systemic absorption and reaches high colonic concentrations, it is hydrolyzed to a less active metabolite, OP-1118. Few studies have completely described critical experimental details of liquid chromatography-tandem mass spectrometry (LC-MS/MS) for quantifying fecal fidaxomicin and OP-1118. This study developed and validated a simple, fast, and sensitive LC-MS/MS method to quantify fidaxomicin and OP-1118 in human and mouse feces. This method simplified fecal sample preparation without the use of solid phase extraction and optimized LC-MS/MS parameters. A broad working range (0.3-1000 ng/ml) in both diluted human and murine fecal matrices was achieved with good intra- and inter-day accuracy (93-107%), precision (1-7%), and recovery (70-105%) as well as little IS-normalized matrix effects. This method was utilized to quantify fidaxomicin and OP-1118 in human and murine fecal samples. This novel method was simple, fast, sensitive, and accurate in analyzing fecal fidaxomicin and OP-1118 and could be deployed to facilitate gut pharmacobiome research.

Feces↗

Pharmacogenetics/genomics and personalized medicine.

Despite the marked advances in drug therapy, some patients do not respond favorably or suffer severe adverse drug effects. Pharmacogenetic studies have shown that polymorphisms of drug metabolizing enzymes, transporters and receptors contribute to variable drug response. Owing to the complexity of drug actions, a broader genomics approach aims at finding new drug targets and optimizing therapy for the individual patient. However, pharmacogenomics has made only a few inroads into clinical practice to date. This review evaluates obstacles that need to be overcome. These include the complexity of mechanisms underlying drug response, given singly or in combination, uncertainty about the genetic underpinnings of complex diseases, such as cancer, diabetes, cardiovascular and mental disorders and a lack of quantitative understanding of the scope of genetic variations, even for well-studied genes. By resolving these hurdles, pharmacogenomics will yield significant, but incremental, therapeutic advances paving the way towards personalized health care.

Drug Therapy, Combination↗

Pathogenesis of alcohol-induced chronic pancreatitis: facts, perceptions, and misperceptions.

The pathogenesis of chronic pancreatitis secondary to chronic alcoholism is not fully understood. A major hurdle in the understanding of the pathogenesis is the inability to study early lesions of the pancreas and the sequential changes. Facts are few; observations are many. Each new hypothesis argues against all previous hypotheses; however, clinical chronic pancreatitis is initiated by one or more of the mechanisms. Good experimental models for alcoholic pancreatitis are not available, limiting the ability to study the pathogenesis. Additional studies on genetic markers and immunologic mechanisms might explain acinar cell injury, which seems to be the earliest lesion in most, if not all, types of chronic pancreatitis. Opie's common channel and obstruction regurgitation theories seem unrelated to chronic pancreatitis. Although biochemical changes of the pancreatic secretion in alcoholic patients promote protein-plug formation, evidence is too weak to consider protein plug as the earliest change. The theory of necrosis of the acinar cell by some unknown mechanism, subsequently leading to fibrosis, is gaining support; however, it is clear that the pathogenesis of alcoholic pancreatitis is not yet fully understood.

Humans↗

B7-H1-targeted immunotherapy for head and neck cancer.

Manipulation of defined costimulatory pathways may provide a means to direct the immune response against head and neck cancer. Three broad hurdles serve as relative obstacles to effective clinical translation of reagents designed to alter costimulatory function. First is an expanding, but still limited, understanding of the biological function of each individual costimulatory pathway. Second is the paucity of reagents suitable for clinical application. Third is the limited number of valid model systems to evaluate the utility of these novel reagents prior to clinical intervention. This review focuses on the role of a recently discovered costimulatory molecule, B7-H1, as a model for the development of novel strategies for immunotherapy of head and neck cancer.

Abatacept↗

Ultrasound-Actuated Gene Editing in Human Kidney Organoids.

Efficient delivery of gene editing ribonucleoproteins (RNPs) into the interior of solid tissues remains a key hurdle to the clinical translation of non-viral CRISPR-Cas9 technologies. Here, we report acoustically-actuated peptide nanoemulsions (NPeps) that can be spatiotemporally guided and activated by ultrasound to ballistically deliver RNPs into cells within the bulk of dense 3D cellular structures. Using human kidney organoids as a model, we demonstrate NPep vectors improve the spatial profile of gene editing in the organoid mass relative to commercial lipofection reagents, without disruption of tissue structure or qualitative viability features. This technologic paradigm is poised to advance imaging-guided, deep tissue RNP delivery modalities to expand the clinical diagnostic and therapeutic potential of CRISPR-Cas9 editing strategies.

Humans↗

Treatment of human disease by adeno-associated viral gene transfer.

During the past decade, in vivo administration of viral gene transfer vectors for treatment of numerous human diseases has been brought from bench to bedside in the form of clinical trials, mostly aimed at establishing the safety of the protocol. In preclinical studies in animal models of human disease, adeno-associated viral (AAV) vectors have emerged as a favored gene transfer system for this approach. These vectors are derived from a replication-deficient, non-pathogenic parvovirus with a single-stranded DNA genome. Efficient gene transfer to numerous target cells and tissues has been described. AAV is particularly efficient in transduction of non-dividing cells, and the vector genome persists predominantly in episomal forms. Substantial correction, and in some instances complete cure, of genetic disease has been obtained in animal models of hemophilia, lysosomal storage disorders, retinal diseases, disorders of the central nervous system, and other diseases. Therapeutic expression often lasted for months to years. Treatments of genetic disorders, cancer, and other acquired diseases are summarized in this review. Vector development, results in animals, early clinical experience, as well as potential hurdles and challenges are discussed.

Dependovirus↗

Stem cell treatment for Parkinson's disease: an update for 2005.

PURPOSE OF REVIEW: The hallmark pathologic feature of Parkinson's disease is loss of melanized dopaminergic neurons within the substantia nigra pars compacta coupled with depletion of striatal dopamine. This is responsible for the major motor features of the disease. Whereas dopaminergic replacement therapy is effective in the early stages of the illness, chronic treatment is associated with motor complications and development of features that do not respond to levodopa therapy. Development of cellular therapies offers the potential to provide more effective treatment for the disease without motor complications. RECENT FINDINGS: Two clinical trials of fetal nigral transplantation failed to meet their primary endpoint and were complicated by the development of dyskinesia that persisted after withdrawal of levodopa ('off-medication' dyskinesia). However, recent studies suggest that both the limited clinical response and off-medication dyskinesia may be related to partial, but incomplete, dopaminergic reinnervation of the striatum and that both might be improved by transplantation of more dopamine neurons. Stem cells offer the potential to provide a virtually unlimited supply of optimized dopaminergic neurons that can provide enhanced benefits in comparison to fetal mesencephalic transplants. Stem cells have now been shown to be capable of differentiating into dopamine neurons that provide benefits following transplantation in animal models of Parkinson's disease. However, cell survival and behavioral responses are limited. There have been numerous advances in enhancing the yield of dopamine neurons from stem cells, and promoting their survival and consequent clinical effects. SUMMARY: Stem cells offer great promise as a therapy for Parkinson's disease, but numerous hurdles remain to be overcome with stem cell therapy. The adverse event profile of transplantation must be determined, and societal and ethical issues addressed. As Parkinson's disease involves degeneration of both dopaminergic and non-dopaminergic neurons, it also remains to be determined if transplantation of even the ideal dopamine neuron will improve non-dopaminergic features of the disease or provide benefits superior to existing therapies.

Animals↗

Morphological aspects of apoptosis in heart diseases.

It has been suggested that apoptosis may be responsible for a significant amount of cardiomyocyte death during acute myocardial infarction as well as for a progressive loss of surviving cells in failing hearts. Typical apoptosis can indeed be induced in cardiomyocytes at the experimental conditions. In actual heart diseases, in contrast, there is very little direct morphological evidence of apoptosis in cardiomyocytes occurring at any stage of myocardial infarction and heart failure, despite the availability of much indirect evidence that includes detection of DNA fragmentation and apoptosis-related factors. For that reason, the potential efficacy of therapeutic intervention to prevent apoptosis remains controversial. This review will survey available data from both animals and humans to critically assess the role of cardiomyocyte apoptosis during myocardial infarction and its relevance to myocardial remodeling and during progression to heart failure. Also considered will be nonmyocyte interstitial cells, which have received less attention than myocytes despite definitive evidence of their apoptosis in the infarcted heart and recent studies suggesting that blockade of apoptosis among these cells mitigates postinfarction cardiac remodeling and heart failure. We conclude from our survey that there are many hurdles to surmount before regulation of apoptosis can be clinically applied in the treatment of myocardial infarction and heart failure.

Animals↗

Microarray technology and gene expression analysis for the study of angiogenesis.

Angiogenesis plays a major role in multiple disease processes including cancer, and new agents that modulate angiogenesis are rapidly entering clinical trials. The understanding of the biological mechanisms and downstream effects for many of these agents is poorly understood. It is therefore important that methods evolve to understand how an agent regulates angiogenesis, in order to promote a higher percentage of successful drug candidates. With the emergence of microarray technology for the evaluation of gene expression, researchers have a powerful tool for dissecting the biological mechanisms of angiogenesis. However, huge data sets and complex statistics pose a hurdle for the investigator to obtain useful and meaningful data. To eliminate problems in data analysis, proper design and planning prior to performing a microarray experiment is crucial to making valid conclusions. This review will discuss the critical factors in designing, performing and analysing microarray experiments, and the utility of various models of angiogenesis for microarray analysis.

Animals↗

Delaying acute graft-versus-host disease in mouse bone marrow transplantation by treating donor cells with antibodies directed at l-selectin and alpha4-integrin prior to infusion.

Acute graft-versus-host disease (GVHD) is still a major hurdle for successful bone marrow transplantation (BMT). Although many immunosuppressive drugs are available, none of them alone or in combination are able to completely abolish acute GVHD. The lifelong immunosuppression profoundly reduces the quality of life of BMT recipients. Therefore, new therapeutic approaches are needed. We previously reported that, in an acute GVHD model using SCID mice as recipient, incubating donor spleen cells with antibodies directed at CD49d and CD62L could significantly delay the occurrence of acute GVHD. To test the potential usefulness of this treatment in BMT, we examined this therapeutic protocol in a mouse BMT model. The present mouse BMT study confirmed our previous results that incubation of donor cells with antibodies directed at CD49d and CD62L prior to infusion into the recipient can effectively delay acute GVHD, allowing the recipients to recover from the side effects of total body irradiation. This one-time treatment is easy and simple and may be modified for clinical usage.

Animals↗

The costs of constraint and prospects for health care reform in Canada.

The sharp decline and equally sharp recovery in public health care spending in the 1990s in Canada set the stage for a broad consideration of reform options but also established hurdles to be overcome in taking action. By moving health care to the center of the federal-provincial agenda, reconfiguring the internal politics of medical and hospital groups, and heightening a public sense of the need for improvement, the legacy of the 1990s prepared the ground for reforms that would "modernize" the Canadian model. But it also yielded a degree of federal-provincial rancor and provider demands for "catch-up," which complicated the process of achieving major change.

Canada↗

Why are systemic glioblastoma metastases rare? Systemic and cerebral growth of mouse glioblastoma.

BACKGROUND: Systemic metastasis of glioblastoma multiforme (GBM) in the form of bulk tumor is rare. This could be because of patient death before clinically detectable systemic metastasis, impediments to systemic egress, or the inability of GBM to grow outside the central nervous system (CNS). In the present paper, we tested this last hypothesis. METHODS: The delayed brain tumor (DBT) cell was characterized with respect to in vitro and in vivo morphology, growth rate, anchorage-independent growth, glial fibrillary acidic protein expression and cytogenetic analysis, and major histocompatibility complex (MHC) typing. We then assayed implantation-induced intracerebral and systemic GBM growth using 3 rodent models with increasing relative immunologic differences between implanted DBT cells and hosts (Balb/c mice, an isograft, MHC I H2, class type D; C3H mice, an allograft; Wistar rats, a xenograft). RESULTS: After implantation in the brain, DBT cells generated tumors that were similar to human GBM. Intracerebral DBT implantation as an isograft or allograft produced only intracranial tumors, whereas intracerebral and systemic implantation as a xenograft produced no tumors. Systemic isograft implantation yielded only systemic tumors. Systemic implantation as allografts produced only transient subcutaneous masses. CONCLUSIONS: Delayed brain tumor cells implanted outside the CNS formed tumors unless there was a significant difference between the immunotype of the implanted cells and host. These results support the hypothesis that the rarity of systemic GBM tumors lies in the presence of physical barriers and/or systemic hurdles that prevent their timely growth. These results also demonstrate that GBMs are antigenic, although not immunogenic, with their syngeneic host. Therefore, GBM may be amenable to targeted immunotherapy given successful artificial priming of the immune system.

Animals↗

Identifying and hurdling obstacles to translational research.

Although there is overwhelming pressure from funding agencies and the general public for scientists to bridge basic and translational studies, the fact remains that there are significant hurdles to overcome in order to achieve this goal. The purpose of this Opinion article is to examine the nature of these hurdles and to provide food for thought on the main obstacles that impede this process.

Animals↗

Use of the nuclease inhibitor aurintricarboxylic acid (ATA) for improved non-viral intratumoral in vivo gene transfer by jet-injection.

BACKGROUND: Stability, integrity and retention of the DNA within the targeted tissue is decisive for efficient gene transfer using naked DNA. Pre-clinical and clinical studies require reproducible transfection rates by preventing rapid degradation of naked DNA in the transduced tissue. Tumor tissues contain nuclease activity, which can affect DNA stability if naked DNA is used. Therefore, inhibition of nuclease-mediated DNA degradation by the nuclease inhibitor aurintricarboxylic acid (ATA) might lead to improved gene transfer efficiency in tumor tissues. METHODS: For both, DNA-degradation analysis and in vivo gene transfer experiments, the beta-galactosidase (LacZ)-expressing pCMVbeta and the cytosine deaminase (CD)-expressing pCMV-CD plasmid were used. Influence of the nuclease inhibitor ATA was determined in tumors, in which naked pCMVbeta or pCMV-CD DNA and ATA was co-administered by jet-injection. The nuclease activity and inhibition by ATA was analyzed using the DNase Alert detection system. The influence of ATA on LacZ expression was determined by specific ELISA and its effect on the therapeutic efficacy of CD gene transfer on tumor growth was determined in vivo. RESULTS: The screening of different human mammary and colon carcinoma models revealed strong nuclease activity rapidly degrading naked plasmid DNA. Co-administration of ATA with pCMVbeta or pCMV-CD for in vivo jet-injection of tumors prevented DNA from nuclease degradation associated with either increased LacZ gene expression or improved reduction in tumor growth. CONCLUSIONS: Tumor-associated nuclease activity is a notable hurdle in gene transfer of naked DNA and therefore inhibition of nucleolytic degradation of plasmid DNA facilitates intratumoral gene expression.

Aurintricarboxylic Acid↗

Transfer of experimental autoimmune thyroiditis by in situ perfusion of thyroids with immune sera.

The role of autoantibodies in the pathogenesis of autoimmune thyroiditis (AT) still remains poorly understood. Serum transfer experiments in experimental AT (EAT) and spontaneous AT (SAT) animal models frequently did not succeed or only resulted in minor thyroid lesions. The inconsistent results may have arisen because of technical problems inherent in serum transfer, the major of which is to obtain high enough concentrations of autoantibodies over long enough periods at the potential site of tissue injury. An attempt was made to circumvent this hurdle by repeated in situ perfusions of the rabbit thyroid via the superior thyroid artery. In situ perfusion of rabbit thyroids with high-titered homologous sera reactive with saline thyroid extract indeed led to thyroiditis characterized by granular deposits of rabbit IgG and C3 in the thyroid follicular basal laminae, cellular infiltrates consisting of mononuclear cells and granulocytes, destruction of the thyroid follicular architecture, and focal fibrosis. Perfusion with control sera lacking thyroid-reactive antibodies did not lead to thyroid lesions. These results demonstrate that humoral antibodies can induce severe AT comparable to actively induced thyroiditis.

Animals↗