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Cystic fibrosis vector gene therapy

Unlimited access to Genetic market reports on 180 countries. Instant industry overview (Market sizing, forecast, key players, trends In Vivo Gene Delivery and Ex Vivo Genetic Engineering for Gene Therapy Development. Advanced Development Platform and Services for Basic and Clinical Research of Gene Therapy Gene Therapy for Cystic Fibrosis Cystic fibrosis is caused by mutations in the gene responsible for producing the cystic fibrosis transmembrane conductance regulator (CFTR) protein. For this reason, scientists are exploring ways to provide a correct copy of the gene to treat CF Since the discovery of the CFTR gene, researchers have been working to develop a gene therapy technique that helps to correct the causative gene in cystic fibrosis patients. Many vector delivery systems have been researched. However, even after years of clinical trials, there is still no FDA approved cystic fibrosis gene therapy technique 1. Curr Gene Ther. 2008 Jun;8(3):201-7. Cystic fibrosis, vector-mediated gene therapy, and relevance of toll-like receptors: a review of problems, progress, and possibilities. Atkinson TJ(1). Author information: (1)Biomedical and Pharmaceutical Consultant, 45 Craignair Road, London, United Kingdom. timatkns@aol.co

Gene therapy

The present invention comprises gene therapy for treating cystic fibrosis(CF). Delivery and expression of a single copy of a normal CFTR gene leads to stable correction of the Cl channel regulation defect present in CF epithelial cells. The present invention includes recombinant viral and plasmid vectors, alternative CFTR gene delivery strategies, and transduced CF cells and cell lines. Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel.Although CF is a multi-organ system disease, most people with CF die of progressive lung disease that begins early in childhood and is characterized by chronic bacterial infection and inflammation

Non-Viral Delivery Systems in Gene Therapy | IntechOpen

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Although the viability of cystic fibrosis (CF) gene transfer to airway epithelium has been demonstrated in vitro and in animal models, so far none of the clinical investigations using adenovirus, adeno-associated virus, lentivirus, cationic lipids or polymers has shown a persistent correction of the ion transport defects that occur in CF Gene Therapy in Cystic Fibrosis Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients After more than two decades since clinical trials tested the first use of recombinant adeno-associated virus (rAAV) to treat cystic fibrosis (CF) lung disease, gene therapy for this disorder has undergone a tremendous resurgence. Fueling this enthusiasm has been an enhanced understanding of rAAV tra

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Cystic fibrosis (CF) is an inherited monogenic disorder, amenable to gene-based therapies. Because CF lung disease is currently the major cause of mortality and morbidity, and the lung airway is readily accessible to gene delivery, the major CF gene therapy effort at present is directed to the lung for CF gene therapy is to design an integrating vector that would lead to persistent and efficient expression of CFTR in the airways. Performing gene therapy experiments is dependent upon a relevant animal model. The CF pig is a large animal model similar in size, anatomy, and physiology to humans Lentiviral (LV) vectors, of the Retroviridae family, show interesting properties for CF gene therapy, since they integrate into the host genome and allow long-lasting gene expression... Since the discovery of the CFTR gene in 1989, many attempts were made to develop a gene transfer system for somatic gene therapy of CF. Cystic Fibrosis (CF) is a recessive disorder, which implies that a single copy of the normal CF gene is sufficient for normal function. Hence, the concept of somatic CF gene therapy is deceptively simple After more than two decades since clinical trials tested the first use of recombinant adeno-associated virus (rAAV) to treat cystic fibrosis (CF) lung disease, gene therapy for this disorder has undergone a tremendous resurgence

Lentiviral vectors are a promising option for CF gene therapy, as they integrate into the host genome and persistently express a transgene of interest. Airway cell tropism can be conferred by pseudotyping. Nonviral vectors such as DNA transposons can also integrate into the genome Cystic fibrosis was really at that moment the prototype disease to be used for gene therapy The CFTR gene was identified in 1989, touching off a wave of enthusiasm for developing a gene therapy cure for the disorder The introduction of a normal copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene into the airway epithelium through gene transfer is an attractive approach to correcting the underlying defects in patients with cystic fibrosis. We tested the feasibility of gene therapy using adenoviral vectors in the nasal epithelium of. A team of researchers affiliated with multiple institutions in Germany has developed a type of gene therapy that could be used to help people with cystic fibrosis. In their paper published in the.

The cystic fibrosis (CF) gene was cloned in 1989 (1), paving the way for gene therapy. Within a year, two studies (2, 3) showed proof-of-principle for correction of the underlying chloride defect in cell lines using viral vectors Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) KB407 is an inhaled therapy that delivers two healthy copies of the CFTR gene directly to patients' lung tissues using a modified version of the herpes simplex virus type 1 (HSV-1). The modified virus is safe for patients. The treatment was found to restore normal CFTR protein production in cultured lung cells in the lab Shortly after the cystic fibrosis (CF) gene was identified in 1989, the race began to develop a gene therapy for this condition. Major efforts utilized full-length cystic fibrosis transmembrane conductance regulator packaged into adenovirus, adeno-associated virus (AAV), or liposomes and delivered to the airways

Gene delivery systems—gene therapy vectors for cystic fibrosis Daniel Klinka, Dirk Schindelhauerb, Andreas Lanerc, Torry Tuckerd, Zsuzsanna Bebokd, Erik M. Schwiebertd, A. Christopher Boyde, Bob J. Scholtea,* aDepartment of Cell Biology, Erasmus MC, Erasmus University, P.O. Box 1738, Rotterdam DR 3000, The Netherlands bLivestock Biotechnology, Life Science Center Weihenstephan, TUM, Freising. The report of the first patients with cystic fibrosis (CF) to receive cystic fibrosis transmembrane conductance regulator gene (CFTR) therapy appeared in 1993, and since then there have been more than 20 clinical trials of both viral and nonviral gene transfer agents.These have largely been single dose to either nose or lower airway and have been designed around molecular or bioelectrical. Due to a natural tropism for the target organ, adenoviral vectors were an obvious first choice for CF gene therapy and the first ever CF gene therapy trial was carried out with an adenoviral vector just a couple of years after cloning the cystic fibrosis transmembrane conductance regulator gene in 1989 In contrast to enthusiasm for AAV gene therapy in other disease fields, relatively few groups strove to overcome the putative shortcomings as a vector for CF gene therapy (10 - 12). Instead, efforts were redoubled by numerous teams to develop alternative vector systems, including lentivirus vectors (13) and nonviral technology (14)

Using RNA therapy makes it easier to control the dose of the therapy, but patients would have to receive treatments more frequently than with other types of gene therapy. MRT5005 is an example of this type of therapy. *** Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice. Section 3: Choosing a Vector for CF Gene Therapy Instructions: A vector is a medium that will be used to deliver the healthy DNA to the cells with unhealthy DNA. A vector could be a virus used to infect the cells with the correct DNA or naked DNA could be applied directly to the cells Discover how you can evaluate the integrity and permeability of ALI cultures in this video. Step-by-step protoco Early gene therapy trials incorporated a normal CFTR gene into the DNA of an adenovirus vector, a vector which injects its DNA into the nucleus of the cell. This vector can successfully express the DNA of a CFTR gene in the cystic fibrosis epithelium using in vivo techniques. 5. These adenovirus vectors proved to be impractical, however, du

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  1. ase deficiency, gene therapy for CF will likely require direct in vivo delivery of gene transfer vectors to the airways of patients with CF
  2. Gene therapy approaches to CF lung disease have demonstrated partial efficacy and short-lived CFTR expression in the airways. Drawbacks in the use of classical gene transfer vectors include immune response to viral proteins or to unmethylated CpG motifs contained in bacterially-derived vector DNA, and shut-off of viral promoters
  3. Abstract: Gene delivery in cystic fibrosis is hampered by extracellular and intracellular biological barriers and inefficient vectors. Although progress is evident, continued bioengineering of DNA, vectors, and delivery technologies will be critical to ensure biocompatibility, safety, and therapeutic effectiveness
  4. FDA approves breakthrough therapy for patients 12+ with cystic fibrosis and at least one F508del mutation in the CFTR gene, estimated at 90% of CF population
  5. Gene therapy for cystic fibrosis (CF) comprises the delivery of a gene for functional cystic fibrosis transmembrane conductance regulator (CFTR) to affected epithelial cells

The idea behind gene therapy for cystic fibrosis is that if we deliver a functional copy of the CFTR gene to lung cells, we would stop progression of the disease. Traditionally gene delivery is done with viruses or liposomes. Viruses have evolved to evade host organism's defensive barriers Cystic Fibrosis - Free download as Powerpoint Presentation (.ppt), PDF File (.pdf), Text File (.txt) or view presentation slides online. A presentation mainly talking about the treatment of cystic Fibrosis. Here, we discuss about diagnosis and how to treat the symptoms of cystic fibrosis as there is still no cure. Also, a new wave of gene therapy involving both viral and non-viral vectors Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in.. Despite significant advances in cystic fibrosis (CF) treatments, a one-time treatment for this life-shortening disease remains elusive. Stable complementation of the disease-causing mutation with a normal copy of the CF transmembrane conductance regulator (CFTR) gene fulfills that goal.Integrating lentiviral vectors are well suited for this purpose, but widespread airway transduction in humans.

Gene Therapy Development - Cystic Fibrosis Gene Therap

Gene Therapy Development for Cystic Fibrosis Cystic fibrosis (CF) is a recessively inherited disease resulting from mutations in the cystic fibrosis trans-membrane conductance regulator (CFTR) gene. Over the last twenty-five years or more, gene therapy has been developed as a novel treatment for CF Here we discuss the history of CF gene therapy, from the discovery of the CFTR gene to current state-of-the-art gene delivery vector designs. While implementation of CF gene therapy has proven more challenging than initially envisioned; thanks to continued innovation, it may yet become a reality The second option, in situ somatic gene therapy, occurs when the vector is placed directly into the affected tissue. This process is being developed for the treatment of cystic fibrosis (by direct infusion of the vector into the bronchi of the lungs), to destroy tumours (eg: brain cancer), and for the treatment of muscular dystrophy

Gene Therapy for Cystic Fibrosis CF Foundatio

Schematic representation of CFTR correction strategies for the treatment of cystic fibrosis. Genetic materials (A) are packaged into a therapeutic vector (B). The therapeutic vector is delivered directly to the patient's lungs (C) or introduced into cells ex vivo(D) Recombinant AAV is an appealing vector system for cystic fibrosis gene therapy. In fact, clinical trials are currently underway using this vector system for the treatment of cystic fibrosis The CF gene is located on the long arm of chromosome 7 [] and, based on the prediction of the amino acid sequence of the encoded protein, it was termed CFTR (cystic fibrosis transmembrane conductance regulator).A whole codon deletion, resulting in the loss of a phenylalanine residue at amino acid position 508 (F508del) in the protein is the commonest mutation, accounting for 50-90% of CF.

Gene therapy is currently being evaluated for a wide range of acute and chronic lung diseases including acute respiratory distress syndrome (ARDS), cancer, asthma, emphysema and cystic fibrosis. Gene Therapy for Cystic Fibrosis What is Cystic Fibrosis? Cystic fibrosis is caused by mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) channel, causing viscous secretions. Clinically this leads to difficulties breathing and frequent lung infections Introduction: Cystic fibrosis (CF) remains a life-threatening genetic disease, with few clinically effective treatment options.Gene therapy and gene editing strategies offer the potential for a one-time CF cure, irrespective of the CFTR mutation class.. Areas covered: We review emerging gene therapies and gene delivery strategies for the treatment of CF particularly viral and non-viral.

Instructions: A vector is a medium that will be used to deliver the healthy DNA to the cells with unhealthy DNA. A vector could be a virus used to infect the cells with the correct DNA or naked DNA could be applied directly to the cells. In this section you will determine what vector will work well for a Cystic Fibrosis patient Gene therapy may offer a potential avenue for curing cystic fibrosis through the repair of the defective CFTR gene. The basic idea behind gene therapy is to deliver a functional copy of the gene to.. An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment, suggest new encouraging results

Cystic Fibrosis Gene Therapy: Vector Systems - The D

Cystic Fibrosis is a genetic condition caused by a faulty gene which a person is born with. However the condition can be diagnosed at any age, most commonly within the first 3 months of being born, from stats by Genetic Disorders Trust (2017) Gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF), suggests new research Under the deal, Boehringer Ingelheim acquires an option to licence IP from Imperial Innovations, the tech transfer arm of Imperial College London, and from gene therapy developer and manufacturer Oxford Biomedica and will collaborate in R&D with the UK Cystic Fibrosis Gene Therapy Consortium, which has a clinical track record of six successful early stage CF gene therapies and has developed a. An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the KU Leuven Laboratory for Molecular Virology and Gene Therapy. Cystic fibrosis or mucoviscidosis is a genetic disorder that makes the mucus [

Cystic fibrosis, vector-mediated gene therapy, and

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 7,1145-1159. Flotte, TR, Carter, BJ (1998) Adeno-associated virus vectors for gene therapy of cystic fibrosis. Methods Enzymol 292,717-732. Flotte TR 2000 Size does matter: overcoming the adeno-associated virus packaging limi Clinical trials of plasmid-based gene therapy exist in phase 1 or 2 for cystic fibrosis, cancer, diabetes, heart disease, hepatitis B, hepatitis C, HIV, human papillomavirus, influenza, and malaria. 35,36 Like any delivery mechanism for gene therapy, plasmids have their downsides. Gene therapy effects delivered by plasmids are often transient. Cystic fibrosis: Gene therapy offers hope to patients after successful trials. Scientists said it was the first time in more than 20 years of research on the cystic fibrosis gene that anyone has. AAV gene therapy has been shown to be successful in other disease areas — I think that really fuelled reinvestigating this vector system for cystic fibrosis Early work with AAV used a form of the virus called AAV2, which isn't very efficient at getting into airway cells

US5240846A - Gene therapy vector for cystic fibrosis

A new partnership in the UK will develop a gene therapy for cystic fibrosis that could treat the disease with a single dose. Cystic fibrosis is a genetic disease that causes mucus to build up in a patient's lungs. As a result, patients suffer from blocked airways and bacterial infections. While treatment advances have increased the life. We have, therefore, built on our non-viral gene therapy experience to develop a new viral vector-based product, which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust

Cystic Fibrosis Gene Therapy: Looking Back, Looking Forwar

Short Lecture on Gene Therapy - Pharma Mirror Magazine

Non-viral vectors in cystic fibrosis gene therapy

Cystic fibrosis (CF) is a genetic disease that hampers the lung function. Despite that the main defective gene has been deeply characterized, some relevant concerns still need to be resolved before considering gene therapy as a realistic medical choice. One of the major issues that need to be strongly considered in order to succeed in the search for an effective gene therapy approach for CF is. Looking for an air-liquid interface culture protocol? Watch our videos to learn how to culture airway epithelial cells at the ALI Keywords: cystic fibrosis; gene therapy; lungs The cystic fibrosis (CF) gene was cloned in 1989 (1), paving the way for gene therapy. Within a year, two studies (2, 3) showed proof-of-principle for correction of the underlying chloride de-fect in cell lines using viral vectors. The next step was to develo A potential gene therapy for cystic fibrosis, KB407, is able to induce the expression of normal CFTR protein in cell models of CF, its developer, Krystal Biotech, reports.. These data are in the poster, In vitro Pharmacology of KB407, an HSV-1 based gene therapy vector, for the treatment of cystic fibrosis (abstract 282, page 131), being presented at the 2020 annual meeting of the.

Cystic Fibrosis Gene Therapy: Vector Systems by Holly

  1. In 2015, the UK Cystic Fibrosis Gene Therapy Consortium took a liposome-based treatment as far as a phase IIb trial 7, but it only improved lung function by a few percentage points and never.
  2. Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy. Hum Gene Ther 2005; 16 : 921-928
  3. Since the cloning of the cystic fibrosis gene (CFTR) in 1989, 18 clinical trials have been carried out, including five in the 2 years reviewed here. Most trials demonstrated proof-of-principle for.
  4. This morning the Cystic Fibrosis Gene Therapy Consortium (GTC) announced the results of clinical trial in 140 patients with cystic fibrosis, which demonstrate the potential for gene therapy to slow - and potentially halt - the decline of lung function in people with the disorder. It is a success that is built on 25 year
  5. The cons of gene therapy include the changes in the gene expression of an individual and how it affects the following generation. Researchers are not confident enough to full understand their employment to make a reliable judgement as to whether the therapy is a proper solution
  6. Gene therapy of cystic fibrosis lung disease need s an efficient delivery and a persistent expression of the CFTR transgene into the airway epithelium. LV vectors appear to be promising vehicles fo
  7. Since the discovery of the genetic basis for cystic fibrosis in 1989, scientists have developed a variety of viral and non-viral vector systems for delivering a corrected CFTR gene back into lung.

Cytokine pattern in cystic fibrosis patients during antibiotic therapy and gene therapy using adenoviral vector. Reix P(1), Bellon G, Bienvenu J, Pavirani A, Levrey-Hadden H. Author information: (1)Service Pneumologie, Allergologie, Mucoviscidose, Hôpital Debrousse, 29, rue Soeur Bouvier, 69322 Lyon Cedex 05, France Creative Biolabs is a world-renowned service provider for cystic fibrosis gene therapy and offers the one-stop, full custom services including the viral and non-viral gene therapy, small RNA and Gene editing for CF, etc. In a word, we are aiming to accelerate our client's gene therapeutic development. Figure 1 Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients. Choice of vector, mode of delivery to airways, translocation of genetic information, and sufficient expression level of the normalized CFTR gene are. EP0549691A1 EP91917139A EP91917139A EP0549691A1 EP 0549691 A1 EP0549691 A1 EP 0549691A1 EP 91917139 A EP91917139 A EP 91917139A EP 91917139 A EP91917139 A EP.

Abstract: Gene therapy is a promising therapeutic modality for the treatment of cystic fibrosis (CF). Despite a better understanding of the molecular organization of the cystic fibrosis transmembrane conductance regulator (CFTR) gene and mutations resulting in pathophysiological and phenotypic alterations, several forms of treatments including gene therapy have failed to yield clinical success Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that encodes a cAMP-regulated anion channel. Although CF is a multi-organ system disease, most people with CF die of progressive lung disease that begins early in childhood and is characterized by chronic bacterial infection and inflammation

Gene Therapy in Cystic Fibrosis - CHES

  1. ate and prevent hereditary diseases such as cystic fibrosis, ADA- SCID etc. It is a possible cure for heart disease, AIDS and cancer. It gives someone born with a genetic disease a chance to life. It can be used to eradicate diseases from the future generations
  2. Gene therapy in cystic fibrosis 1099 www.bjournal.com.br Braz J Med Biol Res 44(11) 2011 advantage of AAV vectors is the fact that they are capabl
  3. The present invention comprises gene therapy for treating cystic fibrosis (CF). Delivery and expression of a single copy of a normal CFTR gene leads to stable correction of the Cl channel regulation defect present in CF epithelial cells. The present invention includes recombinant viral and plasmid vectors, alternative CFTR gene delivery strategies, and transduced CF cells and cell lines.
  4. Request PDF | Update on gene therapy for cystic fibrosis | Cystic fibrosis (CF) is a monogenic autosomal recessive disease. Although several organs are affected, severe lung disease is the cause.
  5. Abstract . Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by mutation of the cystic fibrosis transmembrane conductance regulator (CFTR)
  6. A new adenovirus-based vector (Ad2/CFTR-1) has been constructed in which the cDNA encoding the cystic fibrosis transmembrane conductance regulator (CFTR), the cystic fibrosis (CF) gene product, replaces the early region 1 coding sequences, E1a and E1b. The virus retains the E3 region

MRT5005, currently the most promising gene therapy for cystic fibrosis, is likely to be based on a non-viral vector. Although Translate Bio does not elaborate on their gene delivery technology, the fact that it delivers messenger RNA implies that they use liposomes as vectors Gene therapy for cystic fibrosis Info Japan Prior art keywords cftr cells plj gene vector Prior art date 1990-09-18 Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed. We have, therefore, built on our non-viral gene therapy experience to develop a new viral vector-based product, which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust Introduction: Since the cystic fibrosis (CF) gene was discovered in 1989, researchers have worked to develop a gene therapy. One of the most promising and enduring vectors is the AAV, which has been shown to be safe. In particular, several clinical trials have been conducted with AAV serotype 2

Gene therapy ppUse of Genetically Modified Stem Cells in Experimental

Oxford, UK - 6 August 2018: Oxford BioMedica plc (LSE:OXB) (Oxford BioMedica or the Group), a leading gene and cell therapy group, today announces that it has entered into a process development collaboration agreement with the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations to develop a long-term therapy for patients with cystic fibrosis (CF) Gene Therapy of Cystic Fibrosis Using AAV Fig. 4 - After the optimization of the capsid proteins towards those that most effectively penetrate the nucleus and reach target, a greater amount of variant capsids were observed compared to the starting phenotype, AAV2 CF is one of the most extensively researched genetic diseases as a target for gene therapy development and this can also serve as an important model for gene therapy of other diseases. The aim of gene therapy is to correct the basic defect in CF. This can be achieved by the development of a vector

CF Foundation Arm Awards $3M to Advance Gene Therapy IJMS | Free Full-Text | Vectors for Inhaled Gene Therapy

Gene Therapy Case Study: Cystic Fibrosis Objective: Understand how gene therapy works, what the limitations are and when it is an option. Go to Learn Genetics: Gene Therapy Case Study: Cystic Fibrosis Go through each section (in order) answering the following questions. Paste your answers below or upload them as a word or PDF files Section 1: Understanding the Problem 1 Suggest why people with cystic fibrosis are more likely to suffer from these lung infections than people without cystic fibrosis. [2] Describe the difference between somatic gene therapy and germ line gene therapy. [2] - use a virus as a vector Gene therapy holds promise to cure the disease. Previous studies suggested that the treatment is safe, but largely ineffective for CF patients. However, as gene therapy has recently proven successful for inherited disorders such as haemophilia and congenital blindness, we wanted to re-examine its potential for cystic fibrosis About Cystic Fibrosis and AAV Gene Therapy Affecting more than 30,000 patients in the US and 70,000 worldwide, CF is a genetic disease that impacts multiple tissues, including primarily the lung. Chronic lung infections result in progressive lung damage and premature death Keywords: cystic fibrosis; gene therapy; site-specific gene targeting; viral vector; TALEN 1. Introduction Cystic fibrosis (CF) is an inherited autosomal recessive disease most commonly seen in the Caucasian population [1]. Cystic Fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Sep. 20, 2016 — Gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF), suggests new research. Working with CF pigs, the researchers have.