Claim: Boy with rare disease gets brand new skin with gene therapy.
Verdict: SUPPORTS

Retrieved passages:
title: Genetic engineering
text: (CLL), and Parkinson's disease. In 2012, Alipogene tiparvovec became the first gene therapy treatment to be approved for clinical use. In 2015 a virus was used to insert a healthy gene into the skin cells of a boy suffering from a rare skin disease, epidermolysis bullosa, in order to grow, and then graft healthy skin onto 80 percent of the boy's body which was affected by the illness. Germline gene therapy would result in any change being inheritable, which has raised concerns within the scientific community. In 2015, CRISPR was used to edit the DNA of non-viable human embryos, leading

title: Gene therapy
text: levels of SERCA2, a protein in heart muscles, improving muscle function. The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process. In 2016 it was reported that no improvement was found from the CUPID 2 trial. In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 7–32 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills. The other children had Wiskott-Aldrich syndrome, which leaves them to open to

title: Gene therapy
text: treating children with SCID (severe combined immune deficiency or "bubble boy" disease) from 2000 and 2002, 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 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany. In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. 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

title: Gene therapy
text: infection, autoimmune diseases, and cancer. Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising. In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress. In 2014 a further 18 children with ADA-SCID were cured by gene therapy. ADA-SCID children have no functioning immune system and are sometimes known as "bubble children." Also in October researchers reported that

title: Gene therapy
text: had treated a baby girl, Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]). Children with highly aggressive ALL normally have a very poor prognosis and Layla's disease had been regarded as terminal before the treatment. In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies but that basic research including embryo gene editing should continue. In April the

title: Gene therapy
text: 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results. Clinical trials of gene therapy for sickle cell disease were started in 2014. There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease. In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease. In March

title: Gene therapy
text: Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis and the European Commission approved it in June. This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe. In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the

title: Genetically modified organism
text: rising from adenosine deaminase deficiency (ADA-SCID), although the development of leukemia in some ADA-SCID patients along with the death of Jesse Gelsinger in another trial set back the development of this approach for many years. In 2009 another breakthrough was achieved when an eight year old boy with Leber’s congenital amaurosis regained normal eyesight and in 2016 GlaxoSmithKline gained approval to commercialise a gene therapy treatment for ADA-SCID. As of 2018, there are a substantial number of clinical trials underway, including treatments for hemophilia, glioblastoma, chronic granulomatous disease, cystic fibrosis and various cancers. The most common virus used for gene

title: Gene therapy
text: 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 23-year-old British male, Robert Johnson, in early 2007. 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. Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive

title: Gene therapy of the human retina
text: Gene therapy of the human retina Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV). In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA. These results were widely seen as a success in the gene therapy field, and have generated excitement and momentum for AAV-mediated applications in retinal

title: Gene therapy
text: a newly delivered gene. Similar to organ transplantation, gene therapy has been plagued by this problem. 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

title: Junctional epidermolysis bullosa (medicine)
text: boy's family had fled Syria for Germany in 2013. Upon seeking treatment in Germany, he had lost the epidermis from almost his entire body, with only his head and a patch on his left leg remaining. The group of Italian scientists had previously pioneered a technique to regenerate healthy skin in the laboratory. They used this treatment on the boy by taking a sample from his remaining healthy skin and then genetically modifying the skin cells, using a virus to deliver a healthy version of the LAMB3 gene into the nuclei. The patient underwent two operations in autumn 2015, where

title: Voretigene neparvovec
text: approval of the treatment. The US FDA approved the drug on December 19, 2017. With the approval, Spark Therapeutics received a pediatric disease priority review voucher. The first commercial sale of voretigene neparvovec -- the first for any gene therapy product in the US -- occurred in March 2018. The price of the treatment has been announced at $425,000 per eye. Voretigene neparvovec Voretigene neparvovec (Luxturna) is a novel gene therapy for the treatment of Leber's congenital amaurosis. It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first "in vivo" gene therapy approved by the

title: Severe combined immunodeficiency
text: in ADA SCID and X-linked SCID. In 1990, four-year-old Ashanthi DeSilva became the first patient to undergo successful gene therapy. Researchers collected samples of DeSilva's blood, isolated some of her white blood cells, and used a retrovirus to insert a healthy adenosine deaminase (ADA) gene into them. These cells were then injected back into her body, and began to express a normal enzyme. This, augmented by weekly injections of ADA, corrected her deficiency. However, the concurrent treatment of ADA injections may impair the success of gene therapy, since transduced cells will have no selective advantage to proliferate if untransduced cells

title: 2002 French gene therapy trials
text: implicated in potential treatment for several communicable and non-communicable diseases. SCID was rare in its prevalence and involved a complex mechanism that involved harmful lymphocytic differentiation. The test subjects for this experiment were 11 children of various ages who presented with SCID and were admitted into Necker Hospital. Initially, after the therapy was administered to the children, some showed signs of improved conditions. One child, a three year old, became the face of the experiment's success, as the symptoms of his once life-threatening disease began to diminish. Indeed, this was a breakthrough in the application capabilities of gene therapy in

title: Gene therapy
text: it was still largely an experimental technique. These include treatment of retinal diseases Leber's congenital amaurosis and choroideremia, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia (CLL), acute lymphocytic leukemia (ALL), multiple myeloma, haemophilia, and Parkinson's disease. Between 2013 and April 2014, US companies invested over $600 million in the field. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia. In 2012 Glybera, a treatment for a rare inherited disorder,

title: Gene therapy
text: therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles. Some of the unsolved problems include: 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 1999 because of immune rejection response. One X-SCID patient died of leukemia in 2003. In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy. In 1972 Friedmann and Roblin authored a paper in

title: Christoph Klein
text: By studying rare human diseases, his team is interested in elucidating basic mechanisms orchestrating the development and function of hematopoietic and immune cells. Furthermore, the team strives toward the development of novel cell- and gene-based therapeutic strategies. He discovered numerous diseases and conducted the first clinical gene therapy study for patients with Wiskott Aldrich syndrome. Klein co-founded the international Care-for-Rare Foundation for children with rare diseases. Care-for-Rare is a non-profit organization following the Leitmotiv "From Discovery to Cure". No child should be destined to suffer or die just because his or her disease is very rare, regardless of ethnic origin

title: Gene therapy
text: results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects. In September researchers were able to give trichromatic vision to squirrel monkeys. In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder. An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two

title: Jeans for Genes
text: Programme for care services for children and information and support for families. Funding from the appeal was key in the development of gene therapy at the UCL Institute of Child Health at Great Ormond Street Hospital in London, which has since cured ten children of the life-threatening condition X-linked severe combined immune deficiency X-SCID or 'baby in the bubble' syndrome. The charity also aims to raise awareness and understanding of genetics and what it means to live with a genetic disorder. It provides educational materials for schools through a dedicated website called Genes Are Us Jeans for Genes Jeans for