Research paper on gene therapy


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Why does it matter if we edit them? If those who survive are the fittest, does that also make them the best? And if so, is engineering 'better' babies just evolution, or another step in a long history of eugenics? Once genetic lesions for diseases such as cystic fibrosis and haemophilia were identified, the idea of replacing or correcting defective genes grew into what we now call "gene therapy".

In a completely new approach to treating addiction, researchers use genetically engineered skin cells to inactivate cocaine and block cravings and addiction in mice. An increasing list of rare diseases can now be treated with gene therapy. But we need to figure out a way to make them affordable. New nanoparticles could be an improvement over the current standard — repurposed viruses.

Gene therapy - Latest research and news | Nature

A successful gene therapy for the leading cause of sight loss in children is being reviewed by the FDA. Some scientists are pushing back, placing blame for the unwanted mutations on the experiment, not the technique. Researchers are starting to harness the potential of this much-hyped gene editing technique — with coming applications in medicine, biology and agriculture. A report released by the US National Academies of Science and Medicine underscores the potential of gene editing and acknowledges the sensitivities in managing the ethical dimensions.

Cystic fibrosis CF affects around 3, people in Australia and 70, worldwide.

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It's an inherited disease caused by a mutation in a single gene called CFTR. The delivery of DNA into cells can be accomplished by multiple methods. The two major classes are recombinant viruses sometimes called biological nanoparticles or viral vectors and naked DNA or DNA complexes non-viral methods. In order to replicate , viruses introduce their genetic material into the host cell, tricking the host's cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell.

Scientists exploit this by substituting a virus's genetic material with therapeutic DNA. The term 'DNA' may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well. A number of viruses have been used for human gene therapy, including retroviruses , adenoviruses , herpes simplex , vaccinia , and adeno-associated virus.

Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression , and thus lower therapeutic efficacy. Newer technologies offer promise of solving these problems, with the advent of increased cell-specific targeting and subcellular trafficking control.

Gene therapy

Methods for non-viral gene therapy include the injection of naked DNA, electroporation , the gene gun , sonoporation , magnetofection , the use of oligonucleotides , lipoplexes, dendrimers, and inorganic nanoparticles. More recent approaches, such as those performed by companies such as Ligandal , offer the possibility of creating cell-specific targeting technologies for a variety of gene therapy modalities, including RNA, DNA and gene editing tools such as CRISPR.

Other companies, such as Arbutus Biopharma and Arcturus Therapeutics , offer non-viral, non-cell-targeted approaches that mainly exhibit liver trophism. In more recent years, startups such as Sixfold Bio , GenEdit , and Spotlight Therapeutics have begun to solve the non-viral gene delivery problem. Non-viral techniques offer the possibility of repeat dosing and greater tailorability of genetic payloads, which in the future will be more likely to take over viral-based delivery systems.

Companies such as Editas Medicine , Intellia Therapeutics , CRISPR Therapeutics , Casebia , Cellectis , Precision Biosciences , bluebird bio , and Sangamo have developed non-viral gene editing techniques, however frequently still use viruses for delivering gene insertion material following genomic cleavage by guided nucleases. These companies focus on gene editing, and still face major delivery hurdles. Moderna Therapeutics and CureVac focus on delivery of mRNA payloads, which are necessarily non-viral delivery problems.

Alnylam , Dicerna Pharmaceuticals , and Ionis Pharmaceuticals focus on delivery of siRNA antisense oligonucleotides for gene suppression, which also necessitate non-viral delivery systems.

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In academic contexts, a number of laboratories are working on delivery of PEGylated particles, which form serum protein coronas and chiefly exhibit LDL receptor mediated uptake in cells in vivo. Three patients' deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger , who died in because of immune rejection response. In Friedmann and Roblin authored a paper in Science titled "Gene therapy for human genetic disease?

In a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes. The defective gene of the patient's blood cells was replaced by the functional variant. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated.

She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary. This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena. In Claudio Bordignon , working at the Vita-Salute San Raffaele University , performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.

The success of a multi-center trial for treating children with SCID severe combined immune deficiency or "bubble boy" disease from and , was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in , but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany. Blood was removed from his mother's placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase ADA was obtained and inserted into a retrovirus.

Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew's blood. Injections of the ADA enzyme were also given weekly. After four years more treatment was needed. Jesse Gelsinger 's death in impeded gene therapy research in the US. Trojan Trojan et al.

Sickle-cell disease can be treated in mice. In humans, the use of hydroxyurea to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production. A new gene therapy approach repaired errors in messenger RNA derived from defective genes.

This technique has the potential to treat thalassaemia , cystic fibrosis and some cancers. Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane. In a research team inserted genes into the brain for the first time. They used liposomes coated in a polymer called polyethylene glycol , which unlike viral vectors, are small enough to cross the blood—brain barrier.

If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced. Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus.

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In , it was approved in China for the treatment of head and neck squamous cell carcinoma. In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease , a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system.

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as microRNAs. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene.

In August scientists successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. In November researchers reported on the use of VRX, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial , five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts.

All five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial. In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a year-old British male, Robert Johnson, in early Leber's congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April. In May two more groups reported positive results in independent clinical trials using gene therapy to treat the condition.

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