Bubble boy

The first gene therapy trial was conducted in September 1990. A 4-year-old girl with SCID (an inherited immune disorder disease, otherwise known as the bubble boy syndrome) was treated in Cleveland, Ohio. She is doing well some 10 years after the treatment. A second girl with the same disorder underwent gene therapy and she too continues to do well. 

SCIDGeneTherapy Trial Infantswithsevere combined immunodeficiency disease (SCID, bubble boy syndrome) have a gene defect that leads to a complete lack of white blood cells. Without treatment, these infants die from comphcations of infectious diseases during the first few years of life. The only treatment currently approved for this condition is a bone marrow transplant. 

ADA deficiency ( bubble boy syndrome ) Human clinical trials functional cure... 

The first clinical gene therapy trial began in 1990 for the treatment of adenosine deaminase deficiency. B and T lymphocytes fail to develop in this autosomal recessive disease, resulting in a severe combined immunodeficiency syndrome (SCID) made famous by the bubble boys whose lives were confined to tents in an effort to keep them in a germ-free environment. Only two patients were included in this trial, and although both continued to demonstrate clinical improvement 10 years later, gene therapy did not cure the disease, as investigators had hoped. 

Among the four Jak kinases, Jak-1, Jak-2, Jak-3, and Tyk-2, the yc users all signal via Jak-1 and Jak-3 [30, 31]. This could be explained by the ability of Jak-1 to associate with the chain conferring the specificity for each receptor, namely 1L-2R3 [32, 33], IL-4Ra [34], IL-7Ra [35], and IL-9Ra [36], and probably lL-21Ra [37], whereas Jak-3 associates primarily with yc [33, 35]. The importance of the yc was demonstrated by the discovery that mutations in yc cause X-linked severe combined immunodeficiency (SCID) [30, 38], also named the bubble boy disease. In this disease, both cellular and humoral immunity are defective. In fact, T and NK cells do not develop and even if B cells are present, they are nonfunctional [30, 38]. Interestingly, mutations in Jak-3 were found to cause an autosomal recessive form of SCID [39] and the essential role of Jak-3 in lymphoid development was established [40]. This clearly demonstrated the important role of the Jak-STAT signaling pathway. 

PEG-adenosine deaminase (ADAGEN Enzon) was the first PEGylated protein to enter the market, in 1990 [50]. It is used to treat adenosine deaminase-deficient X-linked severe combined immunodeficiency disease (SCID), commonly known as the bubble boy disease . It is an alternative to bone marrow transplantation and enzyme replacement by gene therapy. Since the introduction of ADAGEN, a large number of PEGylated-protein and -peptide pharmaceuticals have followed (Table 1). 

C. V. Boys, Soap Bubbles and the Forces that Mould Them, Society for Promoting Christian Knowledge, London, 1890 reprint ed.. Doubleday Anchor Books, Science Study Series S3, Doubleday, Garden City, NY, 1959. 

Boys, C.V. Soap Bubbles and the Forces Which Mould Them. New York Doubleday Company, Inc., 1959. 

Boys, C. V., Soap Bubbles, Dover Publications, New York, 1959. 

Reviews of the theory of capillarity and its application to solid-state processes have been written by Herring [1], Mullins [2], and Blakely [3]. Adam wrote a classic text on fluid surfaces [4], For modern mathematical treatments of capillarity, consult Finn s book [5]. For a mathematical treatment of curvature and anisotropic interfaces written for materials scientists, see Taylor s review article [6].1 There are useful analogies between interfaces and phase diagrams which are particularly instructive for materials scientists [7]. Anybody with a milligram of curiosity and a sense of humor must read C.V. Boys s book on soap bubbles although written for children, the book is full of useful insights about the nature of interfaces [8]. 

Under conditions where the weight of the liquid cannot be neglected, the shapes are more complicated. The effect of gravity can, however, be allowed for (see equation (4) below). Plateau s experiments give a very complete experimental proof of the tendency of liquid surfaces to contract the various forms assumed with different supports are of more interest to the mathematician than the physicist, and reference should be made to Plateau s book, Boys s Soap Bubbles,2 or Maxwell s article8 for further details of them. 

A 9 year-old boy was brought to the clinic by his mother. He was complaining of severe pruritus (itching) and bubbles on his arm. 

The Russian peasant of his day never heard of the Periodic Table, but he remembered Dmitri Mendel eft for another reason. One day, to photograph a solar eclipse, he shot into the air in a balloon, flew on a bubble and pierced the sky. And to every boy and girl of the Soviet Union today Mendel eff is a national hero. 

Foam films of different size, shape and spatial orientation are obtained at the approach of individual bubbles or the surfaces of a biconcave drop, or at bubble contact with the solution/air interface, or at withdrawing a frame from a solution, etc. Individual foam bubbles are usually used in the study of foam properties. They prove to be most useful in many cases, for example, in the determination of foam film elasticity, the estimation of gas diffusion from the bubble through the film, the detection of the rupture of the foam bubble films [e.g. 1], Beginning with the remarkable bubbles of Boys [2] and reaching to present day studies, single foam bubbles have since long attracted a considerable interest (see, for instance, the monograph of Dukhin, Kretzschmar and Miller [3]). 

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