Protocol
Effective Targeting of Multiple B-Cell Maturation Antigen–Expressing Hematological Malignances by Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T Cells
B-cell maturation antigen (BCMA) expression has been proposed as a marker for the identification of malignant plasma cells in patients with multiple myeloma (MM). Nearly all MM tumor cells express BCMA, while normal tissue expression is restricted to plasma cells and a subset of mature B cells. Cons... More
Protocol
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-gl... More
Protocol
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID-X1 clinical studies, based on first-generation gammaretroviral vectors, demonstrated good long-term immune r... More
Protocol
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and eff... More
Protocol
Adeno-Associated Virus Gene Therapy for Liver Disease
The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made rema... More
Protocol
Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1
Niemann–Pick disease type C1 (NPC1) is a fatal congenital neurodegenerative disorder caused by mutations in the NPC1 gene, which is involved in cholesterol transport in lysosomes. Broad clinical manifestations of NPC1 include liver failure, pulmonary disorder, neurological deficits, and psychiatric ... More
Primer
Single-Cell RNA Sequencing Upgrades Sensitivity
A new method for DNA amplification and sequencing called TAS-Seq improves the accuracy of single-cell RNA sequencing (scRNA-seq). TAS-Seq uses a terminal transferase enzyme that amplifies and sequences DNA complementary to mRNA (cDNA) in solid phase on a nanowell/magnetic bead-based cell isolation p... More
Protocol
Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan
The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinica... More
Protocol
Advances in Gene Therapy for Diseases of the Eye
Over the last few years, huge progress has been made with regard to the understanding of molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the eye. Such knowledge has led to the development of gene therapy approaches to treat these devastating disorders. Challenges re... More
Protocol
Gene Therapy for Hemoglobinopathies
Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-gl... More
Protocol
High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
Adeno-associated virus (AAV) vectors are quickly becoming the vectors of choice for therapeutic gene delivery. To date, hundreds of natural isolates and bioengineered variants have been reported. While factors such as high production titer and low immunoreactivity are important to consider, the abil... More
Protocol
Effective Targeting of Multiple B-Cell Maturation Antigen–Expressing Hematological Malignances by Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T Cells
B-cell maturation antigen (BCMA) expression has been proposed as a marker for the identification of malignant plasma cells in patients with multiple myeloma (MM). Nearly all MM tumor cells express BCMA, while normal tissue expression is restricted to plasma cells and a subset of mature B cells. Cons... More
Protocol
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-gl... More
Protocol
Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?
More than 20 years ago, X-linked severe combined immunodeficiency (SCID-X1) appeared to be the best condition to test the feasibility of hematopoietic stem cell gene therapy. The seminal SCID-X1 clinical studies, based on first-generation gammaretroviral vectors, demonstrated good long-term immune r... More
Protocol
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by providing a durable therapeutic protein via a single administration. Adeno-associated virus (AAV)-mediated gene transfer is of particular interest as a therapeutic tool because of its safety profile and eff... More
Protocol
Adeno-Associated Virus Gene Therapy for Liver Disease
The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made rema... More
Protocol
Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1
Niemann–Pick disease type C1 (NPC1) is a fatal congenital neurodegenerative disorder caused by mutations in the NPC1 gene, which is involved in cholesterol transport in lysosomes. Broad clinical manifestations of NPC1 include liver failure, pulmonary disorder, neurological deficits, and psychiatric ... More
Protocol
Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan
The development of genome-editing technology could lead to breakthrough gene therapy. Genome editing has made it possible to easily knock out or modify a target gene, while current gene therapy using a virus vector or plasmid hampering modification with respect to gene replacement therapies. Clinica... More
Protocol
Advances in Gene Therapy for Diseases of the Eye
Over the last few years, huge progress has been made with regard to the understanding of molecular mechanisms underlying the pathogenesis of neurodegenerative diseases of the eye. Such knowledge has led to the development of gene therapy approaches to treat these devastating disorders. Challenges re... More
Protocol
Gene Therapy for Hemoglobinopathies
Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-gl... More
Application
A Fast and High Precision Influenza Vaccine Potency Assay
Fast and accurate determination of vaccine titre during influenza vaccine manufacture is important in understanding process performance and correctly scaling each process step. Traditionally assays such as Single Radial Immunodiffusion (SRID) and ELISA have their limitations; SRID, though the ‘gold ... More
Application
A Fast and High Precision Influenza Vaccine Potency Assay
Fast and accurate determination of vaccine titre during influenza vaccine manufacture is important in understanding process performance and correctly scaling each process step. Traditionally assays such as Single Radial Immunodiffusion (SRID) and ELISA have their limitations; SRID, though the ‘gold ... More
Application
Strategies for the Development of a High Throughput Octet® Bio-Layer Interferometry Method to Measure Pharmacokinetics of Monoclonal Antibodies in Preclinical Animal Models
Therapeutic antibodies are developed to treat various diseases, including cancers, immunological disorders, and infectious diseases. The safety and efficacy of these therapeutic molecules may be influenced by the way they interact with the body. Therefore, it is vital to understand how a body reacts... More
Application
Strategies for the Development of a High Throughput Octet® Bio-Layer Interferometry Method to Measure Pharmacokinetics of Monoclonal Antibodies in Preclinical Animal Models
Therapeutic antibodies are developed to treat various diseases, including cancers, immunological disorders, and infectious diseases. The safety and efficacy of these therapeutic molecules may be influenced by the way they interact with the body. Therefore, it is vital to understand how a body reacts... More
Application
Knock-out cell lines and their use in signaling pathways
Knock-out (KO) cell lines are powerful tools to study disease mechanisms, such as the effect of gene disruption on downstream pathways. In this application note, we highlight the effectiveness of Abcam KO cell lines that can be used to investigate signaling pathways, in this case, the TRAIL pathway.... More
Application
Knock-out cell lines and their use in signaling pathways
Knock-out (KO) cell lines are powerful tools to study disease mechanisms, such as the effect of gene disruption on downstream pathways. In this application note, we highlight the effectiveness of Abcam KO cell lines that can be used to investigate signaling pathways, in this case, the TRAIL pathway.... More
Application
Recombinant RabMAb®: Reliable antibodies for reproducible results
Poorly characterized antibodies often fail to provide consistent, reproducible results, thereby wasting materials, researcher time, and money. Compared to traditional polyclonal and monoclonal antibodies, recombinant antibodies exhibit very little batch-to-batch variability, providing scientists wit... More
Application
Recombinant RabMAb®: Reliable antibodies for reproducible results
Poorly characterized antibodies often fail to provide consistent, reproducible results, thereby wasting materials, researcher time, and money. Compared to traditional polyclonal and monoclonal antibodies, recombinant antibodies exhibit very little batch-to-batch variability, providing scientists wit... More
Application
Antibody conjugation guide
Antibody conjugation, also known as antibody labeling, is the process of chemically linking an antibody to a specific tag. There are many common methods used to conjugate antibodies, although they can have complex protocols and usually require a certain level of skill and expertise. In this guide, w... More
Application
Antibody conjugation guide
Antibody conjugation, also known as antibody labeling, is the process of chemically linking an antibody to a specific tag. There are many common methods used to conjugate antibodies, although they can have complex protocols and usually require a certain level of skill and expertise. In this guide, w... More
Primer
Analytical Weights and Balances: Minimizing Errors
Pharma companies that use balances for analytical purposes must check them regularly to ensure they meet required standards and to prevent human errors. The European Directorate for the Quality of Medicines Healthcare (EDQM) has recently published updated guidelines1 for analytical balancing that d... More
Primer
Nanomembrane Technology Rapidly Isolates Exosomes from Tears
Our tears reflect not only our emotions, but they also bear signatures of health and disease.Tears containnanoscale extracellular vesicles called exosomes that carryproteins and nucleic acids released from living cells. However, collecting exosomes from tears is restricted by low recovery, long proc... More
Primer
Fluorination Increases Bioavailability and Stability of Natural Therapeutics
A novel chemoenzymatic method can add fluorine atoms to natural compounds. The method uses an enzyme that acts as a gatekeeper for precursors and designs a pinch of precursor tolerance to create a more promiscuous bacterial enzyme that accepts fluorine-containing reactants.Fluorination of therapeuti... More
Primer
Orthogonal Validation in Genome Editing Discovery Work
Simply put, orthogonal validation uses additional methods that provide very different selectivity to the primary method to confirm or refute a finding. All methods are independent approaches that can answer the same question and are used to evaluate and verify the findings. According toClarence Mill... More
Primer
Solvent Precipitation Method Increases Protein Recovery for Proteomics
Study designs in proteomics aim for inclusivity—to characterize every protein in a sample. However, extracting proteins from complex biological samples involves the use of harsh buffers containing detergents and salts that are incompatible with the proteomics workflow. More proteins are lost in atte... More
Primer
Nanotip Stabilization Enables Prolonged Nanoscale Imaging of Large Samples
An innovation that stabilizes the nanotip used in tip-enhanced Raman spectroscopy (TERS) prolongs the imaging time window well beyond the current 30 minute limit. Scientists have demonstrated that the enhanced system is capable of imaging nanoscale defects over six hours in a micrometer-sized, two-d... More
Primer
Behavior of Metal Bearing Nanomaterials in Biosystems
Nanomaterials are being engineered to facilitate a range of applications in diagnosing diseases and delivering drugs. The widespread adoption of nanotechnological applications in medicine rests on the ability to safely target nanomaterials and understand how they behave in biological tissue environs... More
Primer
Cloning Mice From Freeze Dried Somatic Cells
As species continue to face massive extinction, preserving genetic material through judicious biobanking to enable cloning is key to promoting the survival of species and maintaining biodiversity.Embryos and germ cells are commonly stored at ultra-low temperatures in liquid nitrogen. But storing rep... More
Primer
Rapid Detection of Diverse Infectious Agents in Skin Interstitial Fluid
A rapid and reliable skin-patch test1 can now detect the COVID-19 virus, and potentially other infectious agents in under three minutes, without the need to draw blood. This convenience overcomes a current challenge in identifying infected individuals who are averse to blood tests and could help res... More
Primer
Techniques to Detect Unusual Nucleic Acid Structures
Guanine quadruplexes (G-quadruplexes or G4s) are unusual nucleic acid structures with distinct biological and chemical functions. They have occupied significant attention in the nucleic acid world since they were first identified in 1962 as the basis for the aggregation of 5’-guanosine monophosphate... More
Mixer
Spatial Biology Tools and Techniques
Single-cell spatial phenotyping is transforming biomedical research in fields ranging from oncology to neuroscience. This GEN Protocols Mixer focuses on technological developments and protocols that have enabled spatial approaches in biology. We will explore novel applications of the technology in d... More
Mixer
Soft Skills Needed in the Biotech Workforce
Being a professional in a successful life science company--be it a CRO, a CDMO, a startup, a mid- to medium-sized biotech firm, or an established company with a track record of popular products--requires much more than academic credentials, research expertise and technical knowhow. Soft skills are t... More
Mixer
Organoids for Airway and Alveolar Disease Modeling
In this Mixer, we discussed the development of respiratory airway and alveolar organoids derived from human iPSCs and how these are used in modeling respiratory diseases such as cystic fibrosis. Airway organoid models can be used for testing the therapeutic efficacy of candidate drugs for respirator... More
Mixer
Cell Therapy: Autologous vs Allogeneic Cell Expansion
This Mixer discusses culture systems, integrated consumables, and technical considerations for cell expansion processes in the production of autologous and allogeneic cell therapy products. Our expert panelists will discuss the implementation of scalable, cost-efficient technologies and process opti... More
Mixer
Mapping Tissue Carbohydrates Using Mass Spectrometry
This mixer will be relevant for all interested in methods used in glycoproteomics research that aims to understand the pivotal role of carbohydrate motifs that modify the proteome.Glycoproteomics, a dynamic area of frontier research, provides a systems biology approach to understanding the biologica... More
Mixer
Viral Titer Assays and Interpretations
In this Mixer we will discuss some common methods used to measure viral titers and considerations one should bear in mind when interpreting the results from different assays used to quantify virus particles.Along with all things virology, the recent pandemic has led to focused interest in the accura... More
Mixer
Cell Therapy Development: Tackling Analytic Challenges
Cell therapy products are an exciting new class of medicinal products that offer opportunities to treat and cure diseases that cannot be treated using conventional therapeutic options. The development and manufacturing of cell therapy products pose several challenges compared to other biologics. Yet... More
Video
GEN Protocols Expert Exchanges: Single-Cell RNA Sequencing--Challenges and Solutions
Biological resolution at the level of individual cells is the only route to understanding biological organisms that are a heterogenous composite of different cell types. Single-cell technology is powering the next phase of basic research in biology, translational enquiries, and strategies for person... More
Video
GEN Protocols Expert Exchanges: Nanoparticles in Precision Medicine Diagnostics and Delivery
Particles in the size-range of one to 100 nanometres called nanoparticles exhibit significantly different physical and chemical properties owing to their large surface area to volume ratio. Nanoparticles are finding new applications in medicine. They can combine diagnosis and delivery into one devic... More
Video
GEN Protocols Expert Exchanges: Critical ALS Biomarkers of Neuroinflammation & Oxidative Stress
Biomarkers are essential tools for the development of therapeutics, clinical trial design and patient stratification. Biomarkers also help evaluate the efficacy of therapeutic intervention on disease progression or relapse. Yet, biomarker driven patient stratification, a hallmark in oncology trials,... More
Video
GEN Protocols Expert Exchanges: Patient-Derived Pancreatic Cancer Organoids Predict Response to Therapy
Pancreatic cancer accounts for three percent of all cancers in the United States and is responsible for seven percent of all cancer deaths. Miniature organoids cloned from pancreatic tumors in patients are being grown in laboratories to conduct assays that can predict the effectiveness of chemother... More
Video
GEN Protocols Expert Exchanges: COVID-19 Immunodiagnostic Test Identifies Antibody Source
Need another COVID-19 booster?A new immunodiagnostic test called PictArray helps you decide. The test tells you exactly where your antibodies to the virus are coming from - whether your antibodies are from vaccines or SARS-CoV-2 infections, or both. The test detects SARS-CoV-2 antigens--both spike p... More
Audio
GEN Protocols Expert Exchanges: Next Generation Techniques to Develop Genetic Medicines
The past decade has seen a shift in drug discovery to include targeting the root cause of genetic diseases: loss-of-function, gain-of-function, and change-of-function mutations in the genome. Although first generation genetic medicines like gene therapies heralded a new approach to treating previous... More
Video
GEN Protocols Expert Exchanges: Tools and Techniques to Develop Therapies for Neurological Disorders
The human brain is an exquisitely complex organ, consisting of nearly one hundred billion neurons, an equivalent number of non-neuronal cells and trillions of synapses. Understanding the factors that play a role in the development of the structure and function of the brain is an essential step to de... More
Annual Guide
