• COL7A1
• epidermolysis bullosa
• gene and cell therapy
• gene delivery
• skin
• stem cells
• study outcomes
• treatment

• Humans
• Epidermolysis Bullosa Dystrophica/therapy
• Epidermolysis Bullosa Dystrophica/genetics
• Genetic Therapy/methods
• Cell- and Tissue-Based Therapy/methods
• Collagen Type VII/genetics
• Gene Editing/methods
• Animals
• Keratinocytes/metabolism
• Keratinocytes/transplantation

• 23-15-00433 Russian Science Foundation

• Gene editing
• Human embryonic stem cells
• Human induced pluripotent stem cells
• Loci
• Ribonucleoprotein

• Humans
• Animals
• Mice
• Lung/physiology
• Tissue Engineering/methods
• Respiratory Tract Diseases

• NC/L001039/1 National Centre for the Replacement, Refinement and Reduction of Animals in Research
• MRF_MRF-009-0003-RG-HAITC MRF
• NC/V002384/1 National Centre for the Replacement, Refinement and Reduction of Animals in Research
• MRF_MRF-091-0003-RG-CONFO MRF
• G0802804 Medical Research Council

• cancer treatment
• clustered regularly interspaced short palindromic repeat/CRISPR-associated nuclease 9
• gene editing
• human papillomavirus
• tumor microenvironment

• Biomaterial
• CRISPR
• Drug development
• Morel cultivation
• Mushroom cultivation
• Mycotoxin biosynthesis
• Plastic degradation

• T 1266 Austrian Science Fund FWF
• Mae Fah Luang University
• Basic Research Fund
• Princess Srinagarindra’s Centenary Celebrations Foundation
• National Research Council Thailand
• Czech Academy of Sciences Long-term Research Development Project
• Thailand Research Fund
• Deutscher Akademischer Austauschdienst
• Czech Academy of Sciences
• Chiang Mai University
• STEP Program (CH)
• Kunming Institute of Botany, Chinese Academy of Sciences
• Helmholtz-Zentrum für Infektionsforschung GmbH (HZI) (4214)

The 5-year overall survival rate remains approximately 50% for head and neck (H&N) cancer patients, even though new cancer drugs have been approved for clinical use since 2016. Cancer drug studies are now moving toward the use of three-dimensional culture models for better emulating the unique tumor microenvironment (TME) and better predicting in vivo response to cancer treatments. Distinctive TME features, such as tumor geometry, heterogenous cellularity, and hypoxic cues, notably affect tissue aggressiveness and drug resistance. However, these features have not been fully incorporated into in vitro H&N cancer models. This review paper aims to provide a scholarly assessment of the designs, contributions, and limitations of in vitro models in H&N cancer drug research. We first review the TME features of H&N cancer that are most relevant to in vitro drug evaluation. We then evaluate a selection of advanced culture models, namely, spheroids, organotypic models, and microfluidic chips, in their applications for H&N cancer drug research. Lastly, we propose future opportunities of in vitro H&N cancer research in the prospects of high-throughput drug screening and patient-specific drug evaluation.

• CRISPR
• Knockout
• NPEV
• Poliovirus containment
• RD cells
• WHO-GAP III

Extracellular vesicles (EVs) are secreted by both eukaryotes and prokaryotes, and are present in all biological fluids of vertebrates, where they transfer DNA, RNA, proteins, lipids, and metabolites from donor to recipient cells in cell-to-cell communication. Some EV components can also indicate the type and biological status of their parent cells and serve as diagnostic targets for liquid biopsy. EVs can also natively carry or be modified to contain therapeutic agents (e.g., nucleic acids, proteins, polysaccharides, and small molecules) by physical, chemical, or bioengineering strategies. Due to their excellent biocompatibility and stability, EVs are ideal nanocarriers for bioactive ingredients to induce signal transduction, immunoregulation, or other therapeutic effects, which can be targeted to specific cell types. Herein, we review EV classification, intercellular communication, isolation, and characterization strategies as they apply to EV therapeutics. This review focuses on recent advances in EV applications as therapeutic carriers from in vitro research towards in vivo animal models and early clinical applications, using representative examples in the fields of cancer chemotherapeutic drug, cancer vaccine, infectious disease vaccines, regenerative medicine and gene therapy. Finally, we discuss current challenges for EV therapeutics and their future development.

The genetic modification of stem cells (SCs) is typically achieved using integrating vectors, whose potential integrative genotoxicity and propensity for epigenetic silencing during differentiation limit their application. The genetic modification of cells should provide sustainable levels of transgene expression, without compromising the viability of a cell or its progeny. We developed nonviral, nonintegrating, and autonomously replicating minimally sized DNA nanovectors to persistently genetically modify SCs and their differentiated progeny without causing any molecular or genetic damage. These DNA vectors are capable of efficiently modifying murine and human pluripotent SCs with minimal impact and without differentiation-mediated transgene silencing or vector loss. We demonstrate that these vectors remain episomal and provide robust and sustained transgene expression during self-renewal and targeted differentiation of SCs both in vitro and in vivo through embryogenesis and differentiation into adult tissues, without damaging their phenotypic characteristics.

•

Nanovectors are used to engineer SCs efficiently, safely, and persistently

• DNA vector
• SMAR
• differentiation
• embryonic stem cell
• episome
• induced pluripotent stem cell
• nonintegrative
• reprogramming
• self-renewal
• transgenesis

Background: Type 2 diabetes mellitus is one of the leading causes of morbidity and mortality worldwide and is derived from an accumulation of genetic and epigenetic changes. In this study, we aimed to construct Insilco, a competing endogenous RNA (ceRNA) network linked to the pathogenesis of insulin resistance followed by its experimental validation in patients’, matched control and cell line samples, as well as to evaluate the efficacy of CRISPR/Cas9 as a potential therapeutic strategy to modulate the expression of this deregulated network. By applying bioinformatics tools through a two-step process, we identified and verified a ceRNA network panel of mRNAs, miRNAs and lncRNA related to insulin resistance, Then validated the expression in clinical samples (123 patients and 106 controls) and some of matched cell line samples using real time PCR. Next, two guide RNAs were designed to target the sequence flanking LncRNA/miRNAs interaction by CRISPER/Cas9 in cell culture. Gene editing tool efficacy was assessed by measuring the network downstream proteins GLUT4 and mTOR via immunofluorescence. Results: LncRNA-RP11-773H22.4, together with RET, IGF1R and mTOR mRNAs, showed significant upregulation in T2DM compared with matched controls, while miRNA (i.e., miR-3163 and miR-1) and mRNA (i.e., GLUT4 and AKT2) expression displayed marked downregulation in diabetic samples. CRISPR/Cas9 successfully knocked out LncRNA-RP11-773H22.4, as evidenced by the reversal of the gene expression of the identified network at RNA and protein levels to the normal expression pattern after gene editing. Conclusions: The present study provides the significance of this ceRNA based network and its related target genes panel both in the pathogenesis of insulin resistance and as a therapeutic target for gene editing in T2DM.

• diabetes mellitus
• gene editing
• genes
• insulin resistance
• lncRNA
• miRNA

• Adult
• Aged
• Aged, 80 and over
• CRISPR-Cas Systems
• Case-Control Studies
• Cell Line
• Computational Biology/methods
• Diabetes Mellitus, Type 2/blood
• Diabetes Mellitus, Type 2/genetics
• Female
• Gene Editing/methods
• Gene Expression
• Gene Regulatory Networks
• Hospitals, University
• Humans
• Insulin Resistance/genetics
• Lymphocytes/metabolism
• Male
• MicroRNAs/genetics
• Middle Aged
• RNA, Long Noncoding/genetics
• RNA, Messenger/genetics

• CRISPR
• CRISPR-Cas9 technology
• Cas9
• cancer
• cancer treatment
• lung cancer

Basic and preclinical research founded the progress of personalized medicine by providing a prodigious amount of integrated profiling data and by enabling the development of biomedical applications to be implemented in patient-centered care and cures. If the rapid development of genomics research boosted the birth of personalized medicine, further development in omics technologies has more recently improved our understanding of the functional genome and its relevance in profiling patients’ phenotypes and disorders. Concurrently, the rapid biotechnological advancement in diverse research areas enabled uncovering disease mechanisms and prompted the design of innovative biological treatments tailored to individual patient genotypes and phenotypes. Research in stem cells enabled clarifying their role in tissue degeneration and disease pathogenesis while providing novel tools toward the development of personalized regenerative medicine strategies. Meanwhile, the evolving field of integrated omics technologies ensured translating structural genomics information into actionable knowledge to trace detailed patients’ molecular signatures. Finally, neuroscience research provided invaluable models to identify preclinical stages of brain diseases. This review aims at discussing relevant milestones in the scientific progress of basic and preclinical research areas that have considerably contributed to the personalized medicine revolution by bridging the bench-to-bed gap, focusing on stem cells, omics technologies, and neuroscience fields as paradigms.

• genomics
• induced pluripotent stem cells
• mesenchymal stem cells
• neuroscience
• personalized medicine
• proteogenomics
• proteomics
• stem cells

β-Thalassemias represent a group of genetic disorders caused by human hemoglobin beta (HBB) gene mutations. The radical curative approach is to correct the mutations causing the disease. CRISPR-CAS9 is a novel gene-editing technology that can be used auspiciously for the treatment of these disorders. The study aimed to investigate the utility of CRISPR-CAS9 for gene modification of hematopoietic stem cells in β-thalassemia with IVS-1-110 mutation.

We successfully isolated CD34⁺ cells from peripheral blood of β-thalassemia patients with IVS-1-110 mutation. The cells were transfected with Cas9 endonuclease together with guide RNA to create double-strand breaks and knock out the mutation. The mutation-corrected CD34⁺ cells were subjected to erythroid differentiation by culturing in complete media containing erythropoietin.

CRISPR/Cas-9 is an effective tool for gene therapy that will broaden the spectrum of therapy and potentially improve the outcomes of β-thalassemia.

• CRISPR/Cas-9
• Hematopoietic stem cells
• Hemoglobin beta gene mutation
• Reverse hybridization
• Thalassemia

Thalassemia is a genetic haematological disorder that arises due to defects in the α and β-globin genes. Worldwide, 0.3-0.4 million children are born with haemoglobinopathies per year. Thalassemic patients, as well as their families, face various serious clinical, socio-economic, and psychosocial challenges throughout their life. Different therapies are available in clinical practice to minimize the suffering of thalassemic patients to some extent and potentially cure the disease. Predominantly, patients undergo transfusion therapy to maintain their haemoglobin levels. Due to multiple transfusions, the iron levels in their bodies are elevated. Iron overload results in damage to body organs, resulting in heart failure, liver function failure or endocrine failure, all of which are commonly observed. Certain drugs have been developed to enhance the expression of the γ-gene, which ultimately results in augmentation of fetal haemoglobin (HbF) levels and total haemoglobin levels in the body. However, its effectiveness is dependent on the genetic makeup of the individual patient. At present, allogeneic haematopoietic Stem Cell Transplantation (HSCT) is the only practically available option with a high curative rate. However, the outcome of HSCT is strongly influenced by factors such as age at transplantation, irregular iron chelation history before transplantation, histocompatibility, and source of stem cells. Gene therapy using the lentiglobin vector is the most recent method for cure without any mortality, graft rejection and clonal dominance issues. However, delayed platelet engraftment is being reported in some patients. Genome editing is a novel approach which may be used to treat patients with thalassemia; it makes use of targeted nucleases to correct the mutations in specific DNA sequences and modify the sequence to the normal wild-type sequence. To edit the genome at the required sites, CRISPR/Cas9 is an efficient and accurate tool that is used in various genetic engineering programs. Genome editing mediated by CRISPR/Cas9 has the ability to restore the normal β-globin function with minimal side effects. Using CRISPR/Cas9, expression of BCL11A can be downregulated along with increased production of HbF. However, these genome editing tools are still under in-vitro trials. CRISPR/Cas9 has can be used for precise transcriptional regulation, genome modification and epigenetic editing. Additional research is required in this regard, as CRISPR/Cas9 may potentially exhibit off-target activity and there are legal and ethical considerations regarding its use.

• CRISPR/Cas9
• base editors
• gene therapy
• genome editing
• haematopoietic stem cell transplant
• thalassemia

• CRISPR/Cas9
• Drug discovery
• Gene editing
• Gene therapy
• HIV and SARS-Cov-2 infection
• Human-induced pluripotent stem cells (hiPSCs)

• Animals
• CRISPR-Cas Systems
• Cellular Reprogramming
• Drug Discovery
• Gene Editing
• Genetic Therapy
• Humans
• Induced Pluripotent Stem Cells/cytology
• Induced Pluripotent Stem Cells/metabolism
• Induced Pluripotent Stem Cells/transplantation
• Virus Diseases/genetics
• Virus Diseases/therapy

• CRISPR/Cas9 gene editing
• iPSCs
• recessive dystrophic epidermolysis bullosa
• type VII collagen

• rna interference
• drug delivery systems
• nanoparticle
• rheumatoid arthritis
• osteoarthritis

• Animals
• Arthritis/genetics
• Arthritis/therapy
• Cell Engineering
• Gene Transfer Techniques
• Humans
• Inflammation/pathology
• RNA Interference
• Signal Transduction/genetics

• P30 AR073752 NIAMS NIH HHS
• R00 AR064837 NIAMS NIH HHS
• R01 AR067491 NIAMS NIH HHS
• R01 HL073646 NHLBI NIH HHS
• R01 DK102691 NIDDK NIH HHS
• P30 AR074992 NIAMS NIH HHS

Tissue engineering has yet to reach its ideal goal, i.e. creating profitable off-the-shelf tissues and organs, designing scaffolds and three-dimensional tissue architectures that can maintain the blood supply, proper biomaterial selection, and identifying the most efficient cell source for use in cell therapy and tissue engineering. These are still the major challenges in this field. Regarding the identification of the most appropriate cell source, aging as a factor that affects both somatic and stem cells and limits their function and applications is a preventable and, at least to some extents, a reversible phenomenon. Here, we reviewed different stem cell types, namely embryonic stem cells, adult stem cells, induced pluripotent stem cells, and genetically modified stem cells, as well as their sources, i.e. autologous, allogeneic, and xenogeneic sources. Afterward, we approached aging by discussing the functional decline of aged stem cells and different intrinsic and extrinsic factors that are involved in stem cell aging including replicative senescence and Hayflick limit, autophagy, epigenetic changes, miRNAs, mTOR and AMPK pathways, and the role of mitochondria in stem cell senescence. Finally, various interventions for rejuvenation and geroprotection of stem cells are discussed. These interventions can be applied in cell therapy and tissue engineering methods to conquer aging as a limiting factor, both in original cell source and in the in vitro proliferated cells.

• Aging
• Senescence
• Rejuvenation
• Geroprotection
• Tissue engineering
• Stem cell therapy

We previously reported that CD133, as a putative cancer stem cell marker, plays an important role in cell proliferation and invasion in colon cancer. To understand the role of CD133 expression in colon cancer, we evaluated the inhibitory effect of CD133 in colon cancer cells. In this study, we generated CD133^knockout colon cancer cells (LoVo) using the CRISPR-Cas9 gene editing system. CD133⁺ colon cancer cells (LoVo) were infected with the lentiviral vector carrying CD133 gRNA and purified cell by culturing single cell colonies. CD133^knockout cells was validated by western blot and flow cytometry analysis. In functional study, we observed a significant reduction in cell proliferation and colony formation in CRISPR-Cas9 mediated CD133 ^knockout cells in compare with control (P < 0.001). We also found the anticancer effect of stattic was dependent on CD133 expression in colon cancer cells. Although CD133^knockout cells could not completely block the tumorigenic property, they showed remarkable inhibitory effects on the ability of cell migration and invasion (P < 0.001). In addition, we examined the epithelial mesenchymal transition (EMT)-related protein expression by western blot. The result clearly showed a loss of vimentin expression in CD133^knockout cells. Therefore, CRISPR-Cas9 mediated CD133^knockout can be an effective treatment modality for CD133⁺ colon cancer through reducing the characteristics of cancer stem cells.

• AC133 Antigen/deficiency
• AC133 Antigen/genetics
• AC133 Antigen/pharmacology
• CRISPR-Cas Systems/genetics
• Cell Proliferation
• Colonic Neoplasms/genetics
• Colonic Neoplasms/pathology
• Colonic Neoplasms/therapy
• Cyclic S-Oxides/pharmacology
• Epithelial-Mesenchymal Transition/genetics
• Gene Knockout Techniques/methods
• Humans
• Neoplasm Invasiveness/prevention & control
• Neoplastic Stem Cells
• Tumor Cells, Cultured
• Vimentin/metabolism

• Developmental block
• Energy substrates
• Hamster embryo culture medium (HECM)
• Hatching
• One-cell embryo
• Viability
• Vitamins

• 3D spheroids
• Autologous chondrocyte implantation
• CRISPR-Cas9
• Cartilage
• MMP13

• Cell differentiation
• Innovative therapies
• Pancreatic organoids
• Stem cells
• Type 1 diabetes mellitus

• Animals
• Cell Differentiation
• Diabetes Mellitus, Type 1/pathology
• Diabetes Mellitus, Type 1/therapy
• Humans
• Islets of Langerhans/cytology
• Islets of Langerhans Transplantation/methods
• Organoids/cytology
• Regenerative Medicine/methods
• Stem Cells/cytology

• CRISPR
• Cas9
• Gene editing
• Knockin
• Knockout
• Pluripotent stem cells
• Trex2
• i53

• Animals
• CRISPR-Cas Systems
• Cardiotoxicity/genetics
• Cardiotoxicity/therapy
• Cardiovascular Diseases/genetics
• Cardiovascular Diseases/therapy
• Cell Differentiation/genetics
• Gene Editing
• Genome, Human
• Humans
• Myocytes, Cardiac/cytology
• Myocytes, Cardiac/physiology
• Stem Cells/cytology
• Stem Cells/physiology

One of the fundamental discoveries in the field of biology is the ability to modulate the genome and to monitor the functional outputs derived from genomic alterations. In order to unravel new therapeutic options, scientists had initially focused on inducing genetic alterations in primary cells, in established cancer cell lines and mouse models using either RNA interference or cDNA overexpression or various programmable nucleases [zinc finger nucleases (ZNF), transcription activator-like effector nucleases (TALEN)]. Even though a huge volume of data was produced, its use was neither cheap nor accurate. Therefore, the clustered regularly interspaced short palindromic repeats (CRISPR) system was evidenced to be the next step in genome engineering tools. CRISPR-associated protein 9 (Cas9)-mediated genetic perturbation is simple, precise and highly efficient, empowering researchers to apply this method to immortalized cancerous cell lines, primary cells derived from mouse and human origins, xenografts, induced pluripotent stem cells, organoid cultures, as well as the generation of genetically engineered animal models. In this review, we assess the development of the CRISPR system and its therapeutic applications to a wide range of complex diseases (particularly distinct tumors), aiming at personalized therapy. Special emphasis is given to organoids and CRISPR screens in the design of innovative therapeutic approaches. Overall, the CRISPR system is regarded as an eminent genome engineering tool in therapeutics. We envision a new era in cancer biology during which the CRISPR-based genome engineering toolbox will serve as the fundamental conduit between the bench and the bedside; nonetheless, certain obstacles need to be addressed, such as the eradication of side-effects, maximization of efficiency, the assurance of delivery and the elimination of immunogenicity.

Induced pluripotent stem cells (iPSCs) have provided new opportunities to explore the cell biology and pathophysiology of human diseases, and the lysosomal storage disorder research community has been quick to adopt this technology. Patient-derived iPSC models have been generated for a number of lysosomal storage disorders, including Gaucher disease, Pompe disease, Fabry disease, metachromatic leukodystrophy, the neuronal ceroid lipofuscinoses, Niemann-Pick types A and C1, and several of the mucopolysaccharidoses. Here, we review the strategies employed for reprogramming and differentiation, as well as insights into disease etiology gleaned from the currently available models. Examples are provided to illustrate how iPSC-derived models can be employed to develop new therapeutic strategies for these disorders. We also discuss how models of these rare diseases could contribute to an enhanced understanding of more common neurodegenerative disorders such as Parkinson’s disease, and discuss key challenges and opportunities in this area of research.

Summary: This Review discusses how induced pluripotent stem cells (iPSCs) provide new opportunities to explore the biology and pathophysiology of lysosomal storage diseases, and how iPSCs have illuminated the role of lysosomes in more common disorders.

• Gaucher disease
• IPSC models
• Lysosomal enzymes
• Lysosomal storage disorders
• Neurodegeneration

The rising interest in human induced pluripotent stem cell (hiPSC)-derived organoid culture has stemmed from the manipulation of various combinations of directed multi-lineage differentiation and morphogenetic processes that mimic organogenesis. Organoids are three-dimensional (3D) structures that are comprised of multiple cell types, self-organized to recapitulate embryonic and tissue development in vitro. This model has been shown to be superior to conventional two-dimensional (2D) cell culture methods in mirroring functionality, architecture, and geometric features of tissues seen in vivo. This review serves to highlight recent advances in the 3D organoid technology for use in modeling complex hereditary diseases, cancer, host–microbe interactions, and possible use in translational and personalized medicine where organoid cultures were used to uncover diagnostic biomarkers for early disease detection via high throughput pharmaceutical screening. In addition, this review also aims to discuss the advantages and shortcomings of utilizing organoids in disease modeling. In summary, studying human diseases using hiPSC-derived organoids may better illustrate the processes involved due to similarities in the architecture and microenvironment present in an organoid, which also allows drug responses to be properly recapitulated in vitro.

• 3D organoids
• CRISPR/Cas-9
• disease modeling
• drug screening
• genome editing
• hereditary diseases
• human induced pluripotent stem cells
• infectious diseases
• microenvironment
• neurodevelopmental disorders

Genetic variations play an important role in the clinical presentation and progression of Alzheimer’s disease (AD), especially early-onset Alzheimer’s disease. Hundreds of mutations have been reported with the majority resulting from alterations in β-amyloid precursor protein (APP), presenilin 1 (PSEN1), or presenilin 2 (PSEN2) genes. The roles of these mutations in the pathogenesis of AD have been classically confirmed or refuted through functional studies, where the mutations are cloned, inserted into cell lines, and monitored for changes in various properties including cell survival, amyloid production, or Aβ42/40 ratio. However, these verification studies tend to be expensive, time consuming, and inconsistent. Recently, the clustered regularly interspaced short palindromic repeats-CRISPR-associated protein 9 (CRISPR–Cas9) system was developed, which improves sequence-specific gene editing in cell lines, organs, and animals. CRISPR–Cas9 is a promising tool for the generation of models of human genetic diseases and could facilitate the establishment of new animal AD models and the observation of dynamic bioprocesses in AD. Here, we recapitulated the history of CRISPR technology, recent progress, and, especially, its potential applications in AD-related genetic, animal modeling, and functional studies.

• Alzheimer’s disease
• Aβ42/40 ratio
• CRISPR–Cas9
• mutation

Scientists have tried to reprogram various origins of primary cells into human induced pluripotent stem cells (hiPSCs). Every somatic cell can theoretically become a hiPSC and give rise to targeted cells of the human body. However, there have been debates on the controversy about the differentiation propensity according to the origin of primary cells. We reprogrammed hiPSCs from four different types of primary cells such as dermal fibroblasts (DF, n = 3), peripheral blood mononuclear cells (PBMC, n = 3), cord blood mononuclear cells (CBMC, n = 3), and osteoarthritis fibroblast-like synoviocytes (OAFLS, n = 3). Established hiPSCs were differentiated into chondrogenic pellets. All told, cartilage-specific markers tended to express more by the order of CBMC > DF > PBMC > FLS. Origin of primary cells may influence the reprogramming and differentiation thereafter. In the context of chondrogenic propensity, CBMC-derived hiPSCs can be a fairly good candidate cell source for cartilage regeneration. The differentiation of hiPSCs into chondrocytes may help develop “cartilage in a dish” in the future. Also, the ideal cell source of hiPSC for chondrogenesis may contribute to future application as well.

• CRISPR
• engineered nuclease
• genome engineering
• plant biotechnology

• CRISPR/Cas9
• Cancer therapy, combination therapy
• Cisplatin
• Delivery vehicle
• Exosomes
• Gene editing
• PARP-1

• Animals
• Antineoplastic Agents/therapeutic use
• CRISPR-Cas Systems
• Cell Line, Tumor
• Cisplatin/therapeutic use
• Combined Modality Therapy
• Cytokines/immunology
• Exosomes
• Female
• Gene Transfer Techniques
• Genetic Therapy
• HEK293 Cells
• Humans
• Leukocytes, Mononuclear/immunology
• Mice, Inbred BALB C
• Mice, Nude
• Neoplasms/genetics
• Neoplasms/pathology
• Neoplasms/therapy
• Poly (ADP-Ribose) Polymerase-1/antagonists & inhibitors
• Poly (ADP-Ribose) Polymerase-1/genetics
• RNA/genetics

• Gene correction
• Huntington’s disease
• Induced neural stem cells
• Stem cell-based therapies
• Transplantation