Analysis of the protein-coding regions of the genome can help reveal genetic influences on disease and population health. Our company is committed to establishing cutting-edge sequencing platforms to provide customers with the best rare disease whole-exome sequencing (WES) services. Our complete bioinformatics solutions help you explore the pathogenic mechanisms of rare diseases and find causative and susceptibility loci that are significantly associated with the disease.

Pharmacokinetics mainly studies the movement of drugs in the body. Our company has a group of experienced and high-level scientists who are good at pharmacokinetic studies. We can provide a complete set of in vivo and in vitro pharmacokinetic study services from experimental design, implementation, and analysis, and provide customers with a full range of pharmacokinetics services to accelerate your rare disease therapy development.

The improvement of exercise endurance is the most direct manifestation of the strengthening of fatigue resistance. The loaned swimming test reflects the degree of exercise fatigue in rodents by detecting the length of swimming time. This experiment is the most direct and objective index to evaluate the anti-fatigue effect of drugs. Our company is dedicated to providing efficient one-stop services for projects and scientific research related to the field of anti-fatigue drug screening and action studies. We provide our clients with complete solutions for professional rodent loaned swimming test, helping them to obtain scientifically reliable experimental results in their relevant studies.

When drugs are absorbed by the human body, the molecular size and structure, the permeability of the gastrointestinal membrane, etc. affect the degree and rate of absorption. After absorption, it is transported from the blood to various tissues and organs. The study of the distribution in the body helps to understand the dosage and form of the drug. Afterward, the drug is metabolized by the body, and common types of reactions are oxidation, hydrolysis, reduction, and conjugation. The ultimate goal of metabolism is to facilitate the excretion of drugs from the body, otherwise, accumulation of the drug in the blood will lead to toxicity.

Animal Models for Retinal Degeneration Orphan diseases characterized by retinal degeneration include porcine psychogenic retinitis, LCA, and Stargardt's disease. Their etiology is closely related to one or more underlying genetic defects that cause photoreceptor dysfunction. Our team of experts provides clients with a variety of animal models for the development of therapies for rare retinal degenerative diseases.

The gait analysis tests are mainly completed by a gait analysis system using the footprint bright refraction technology. In the gait analysis experiment, rodents walk from one side of the walking platform to the other. During the process, a high-speed camera located under the walking platform quickly captures the footprints of the experimental animals and the differences in foot pressure, allowing for a quantitative assessment of the rodents' locomotion by analyzing their footsteps and gait. Our company' gait analysis system software can process video data. We calculate various parameters based on the size, position, gait dynamics and pressure of each footstep of the experimental animal to provide qualitative and quantitative analysis of footsteps and gait for our customers.

To date, viral and non-viral vectors have been widely used as two common strategies for delivering genes of interest to multiple target tissues, and they have been successfully used to treat a variety of genetic diseases such as cystic fibrosis, Leber's congenital amaurosis, and various severe combined immunodeficiency (SCID). Viral vectors are highly efficient at transducing genes but are immunogenic. Non-viral vectors have lower transfection efficiency, but they are generally less immunogenic. Both types of vectors have advantages and disadvantages that hinder their therapeutic endpoints in clinical trials. To take advantage of the strengths of both types of vectors, researchers have attempted to develop hybrid vector combinations of viral and chemical vectors to achieve higher gene delivery efficiency than individual vectors alone. These hybrid vectors overcome the limitations associated with both delivery systems while enhancing desired features such as low immunogenicity, targe

Retroviruses are single-stranded RNA viruses capable of converting RNA to cDNA by the action of reverse transcriptase, which is then amplified by the action of proteases such as DNA replicase, transcriptase, and translatorase. Researchers usually design replication-defective viruses based on certain properties of retroviruses to produce expression vectors capable of carrying specific target genes, known as retroviral vectors. Retroviral vectors are currently the most widely used method for human gene transfer due to their relatively high efficiency of gene transfer and their ability to randomly insert and stably integrate exogenous genes into the host cell genome for sustained expression. Among these, lentiviral vectors and gamma-retroviral vectors have been widely used in gene therapy, for example, to introduce Chimeric Antigen Receptor (CAR) genes into T cells for the targeted killing of tumors by CAR-T cells. Gene therapy has gradually replaced currently available therapies for r

The diagnosis and treatment of rare diseases have long been a daunting challenge due to their complex and often undefined nature. However, with recent advancements in technology, specifically in the realm of genomics, whole genome sequencing (WGS) has emerged as a powerful tool that has revolutionized the diagnosis and management of rare diseases. This groundbreaking technique offers hope to countless individuals and their families who have suffered from the uncertainties and lack of answers associated with these conditions. What are rare diseases? Rare diseases are a diverse group of disorders that affect a small percentage of the population, often occurring as a result of genetic mutations. With over 7,000 rare diseases identified worldwide, their rarity makes them particularly challenging to diagnose accurately. Traditional diagnostic methods often involve time-consuming and costly procedures, such as multiple invasive tests, leading to delayed or missed diagnoses. Advances in

CNTs are a new type of nanomaterial that is attracting attention as gene therapy vectors due to their unique advantages such as large aspect ratio, good biocompatibility, superior mechanical properties, and ease of functionalization. Despite their promising advantages, the use of primitive CNTs in biomedical applications has been limited by their highly hydrophobic surface, which results in poor water solubility and dispersion. Recent studies have shown that CNTs can be surface modified or functionalized using a variety of physicochemical means, making them a new and ideal gene transfer vector. CNTs can be used as excellent carriers for plasmid DNA, small interfering RNA (siRNA), and microRNA with different types of functionalized modifications (e.g. polycations).

Gene therapy can be adapted to each individual to treat a wide range of diseases including cancer and rare diseases. The development of suitable gene transfer vectors to introduce exogenous genes into target cells is of great importance. Currently, research in gene therapy vectors has developed from viral vectors such as retroviruses, adenoviruses, and adeno-associated viruses to today's novel vectors such as baculovirus vectors, which provide a relatively safe, scalable, and cost-effective gene transfer strategy for gene therapy. The baculovirus gene delivery system is an easily modifiable gene therapy system that has been reported to enable site-specific delivery, mitigate adverse effects and improve therapeutics, with the potential to be the cost-effective and efficient backbone for gene therapy. Although challenges remain for recombinant baculoviruses in gene therapy, their high DNA-carrying capacity, maneuverability, and ability to express multiple exogenous genes simultaneousl

Viral vectors have shown great promise in various areas of gene therapy and have led to major breakthroughs in the development of therapies for rare diseases such as severe combined immune deficiency (SCID-X) and hemophilia. Currently, adeno-associated virus (AAV), adenovirus, and lentiviral vectors are frequently used. However, the less commonly used alphavirus vectors offer interesting properties for gene therapy applications and are expected to contribute to the development of gene therapies for rare cancers. Alphaviruses are a class of RNA viruses that can replicate in large numbers within the cytoplasm of the host cell. The replacement of structural protein genes with exogenous genes in an alphavirus vector still has many of the biological properties of an alphavirus such as a broad host spectrum of infection, the ability to self-replicate, and induce apoptosis in transfected cells, while being able to express exogenous genes in large numbers and not integrating easily with the

Why need viral vector characterization? Cell and gene therapies for various rare diseases are currently undergoing clinical trials worldwide. The rapid development in this field has led to an increase in regulatory scrutiny and product characterization requirements, as well as a bottleneck in viral vector supply. The manufacturing processes and analytical tools for gene therapy viral vectors need to be continuously improved to meet these demands. Viral vector characterization covers a wide range of viral vectors, such as lentiviral (LV) vectors, adenoviral vectors, and adeno-associated viral (AAV) vectors. Sustained improvements and the establishment of rapid and robust viral vector characterization techniques are crucial for stringent quality control, in order to increase the likelihood of successful development of the required gene therapies. The high complexity of viral vectors is one of the challenges in their characterization and quality control testing. Establishing accurate,

What is a rare disease? A rare disease, also known as an orphan disease, is a medical condition that affects a small number of people. In most countries, a disease is considered rare when it affects fewer than 1 in 2,000 individuals. However, this definition may vary from country to country. Rare diseases can be genetic, meaning they are caused by an alteration or mutation in a person's genes, or they can be acquired later in life due to infections, environmental factors, or other causes. These conditions are often chronic, progressive, and potentially life-threatening. They can have a wide range of symptoms and affect various organs and systems in the body. Examples of rare diseases 1. Hutchinson-Gilford progeria syndrome: This rare genetic disorder causes rapid aging in children, resulting in a variety of health issues. 2. Fibrodysplasia ossificans progressiva: A condition in which soft tissues progressively turn into bone, resulting in loss of mobility and a second skeleton

Characterization of plasmids or nucleic acid constructs is fundamental to the development of gene therapy approaches. We are committed to providing automated and highly sensitive analytical methods to fully measure and characterize nucleic acids and plasmids, which are essential for the discovery and manufacture of gene therapies for rare diseases. Our transferable and reproducible workflows can provide the accuracy you need and save you time. Background Gene therapy is an emerging therapeutic modality that treats diseases by manipulating the expression of genetic material. It has significant potential for curing rare diseases that cannot be cured by conventional therapies. The process of gene therapy varies depending on the in vivo and in vitro routes, but regardless of the type of gene therapy approach, the construction of a plasmid vector, i.e., the insertion of the target gene into the plasmid by means of genetic engineering, using principles such as recombination, is the most fu

In many cases, gene therapy requires a vector to deliver the gene therapy drug to the target cell. Viral vectors have been one of the most widely studied vectors due to their outstanding transduction efficiency and other significant advantages. Viral vector-based gene therapy has now achieved good clinical results. More than a dozen viral gene therapy products have been approved for the treatment of cancer, infectious diseases, and rare single-gene disorders. The main types of viral vectors used clinically for in vivo gene therapy include adenovirus, adeno-associated virus (AAV), herpes simplex virus (HSV), retroviruses, and lentiviruses. In recent years, scientific advances in viral vector engineering, rare disease genome identification, and gene editing are ushering in a new era of viral gene therapy. Rare disease research institutions are committed to using cutting-edge technologies in molecular biology and cell biology to provide comprehensive viral vector development solutions,