Other Names: Enfuvirtide, T-20.

Makers: Trimeris and Roche.

Disease Treated: HIV-1 infection in treatment-experienced patients with evidence of HIV-1 replication despite ongoing antiretroviral therapy.

Approval Status: FUZEON was approved by the U.S. FDA on March 13, 2003.

Chemical/Biological Nature: FUZEON is a synthetic, linear peptide comprised of 36 naturally occurring amino acids. Its N-terminus is acetylated and its C-terminus is a carboxamide. It has a molecular weight of 4,492 daltons.

Administration: 90 milligrams of FUZEON in a 1ml volume is administered by subcutaneous injection twice a day.

Mechanism of Action: FUZEON is the first fusion inhibitor, representing the first new class of anti-HIV treatments ... Read more

The ends of linear eukaryotic chromosomes are capped by telomeres, which consist of tandem repeats of TTAGGG. In human cells, the overall telomere length ranges from 15-20kb at birth, to less than 5kb in some individuals with chronic diseases. Telomeres ensure the integrity and stability of chromosomes. Each time a cell divides, the telomere is shortened a bit until it reaches a minimum length at which cells stop dividing and remain quiescent or die by triggering a p53- or RB (retinoblastoma)-mediated DNA damage response.

Telomere length is regulated by telomerase, the enzyme that adds the hexameric repeat TTAGGG back ... Read more

Lentiviral vectors have been proven to be the most efficient means for stable gene delivery into primary human cells, including hematopoietic stem cells and embryonic stem cells. Progress has been made in recent years to enhance their safety and efficiency. The most significant modification is to give vectors a self-inactivating feature. Another area that has witnessed considerable progress is the use of lentiviral vectors to deliver small interfering RNA into mammalian cells.

Traditionally, vectors derived from the Moloney murine leukemia virus were most widely used for gene transfer. Unfortunately, there is a significant limitation: such vectors require cell division to integrate ... Read more

In the last decade and especially in recent years, several classes of peptides have caught the attention of researchers for their ability to penetrate the cell membrane with dexterity. It is a natural progression to envisage the potential of these peptides to be tools for delivering molecules into cells. Despite its mechanism not being well understood, the peptide-mediated membrane translocation may be a promising tool where traditional delivery methods are not up to the task.

The most well known cell-permeable peptide is the signal peptide that is conjugated to most newly synthesized membrane-bound or secreted proteins, such as those destined for ... Read more

The completion of the Human Genome Project sparked the emergence of new technologies in biomedical research for functional studies. Microarrays, which consist of ordered and immobilized capture groups paired with a variety of detection groups, are among the rapidly developing technologies. DNA microarrays have been devised to study genome functions by profiling mRNA levels in normal and disease conditions. On the other hand, protein microarrays have emerged to reveal genes at the functional level. Antibody microarrays have appeared in the recent literature as a special group in protein microarrays. The numerous monoclonal antibodies accumulated in the last 20 years provide ... Read more

Prenatal evaluation of certain fetal genetic traits in high-risk pregnant women is a routine part of modern obstetric care. Many of the current methods rely on invasive methods such as amniocentesis and chorionic villus sampling that are associated with an inherent risk of fetal loss. Noninvasive approaches to prenatal diagnosis are greatly desired.

In recent years, scientists have discovered that fetal cells, cell-free fetal DNA and cell-free fetal mRNA are circulating in maternal blood with considerable turnover rates. They present unique opportunities for evaluating prenatal genetics noninvasive to fetuses.

Fetal cells recovered from maternal blood provide the only source of ... Read more

There are well-documented genetic variations in a patient’s response to drugs. The response includes both drug efficacy and the adverse reactions. The success rate of drugs is about 70% with variable efficacy due to the genetic variation of the drug’s target protein. For example, about 30% of patients treated with statins (Lipitor, etc.), 35% of patients treated with β-blockers (Propranolol, etc.), and 50% of patients treated with tricyclic antidepressants do not respond to the treatment.

Pharmacogenetics (or pharmacogenomics when studied through genome-wide approaches) investigates the role of inheritance in the individual’s variation in response to a drug. It identifies ... Read more

Other Names: Agalsidase beta.

Maker: Genzyme.

Disease Treated: Fabry disease, which is an X-linked hereditary disorder of glycosphingolipid metabolism inflicting about 5,000 people, mostly men, world-wide. Without treatments, Fabry patients have an average life expectancy of 50 years.

Approval Status: FABRAZYME was approved by the U.S. FDA on April 24, 2003. It is the first drug approved for Fabry disease, which was designated as an Orphan Disease.

Chemical/Biological Nature: FABRAZYME is the same as the naturally occurring enzyme a-galactosidase A. It is produced by recombinant DNA technology in chinese hamster ovary cells. It has 398 amino acids and has a molecular weight of approximately ... Read more

Other Names: Pegvisomant, B2036-PEG.

Maker: Pharmacia (Pfizer).

Disease Treated: Acromegaly, in patients who had an inadequate response to current therapies or for whom the current available therapies are not appropriate. It is estimated that the number of patients diagnosed with acromegaly in the U.S., Europe, and Japan is in the tens of thousands.

Approval Status: SOMAVERT was approved by the U.S. FDA on March 26, 2003.

Administration: 10mg of SOMAVERT is administered by subcutaneous injection daily. Prior to a normal course of treatment, a loading dose of 40mg of SOMAVERT should be given to the patients with physician supervision. Serum level of IGF-1 should ... Read more

Other Names: Teriparatide [rDNA origin].

Maker: Eli Lilly Inc.

Disease Treated: Osteoporosis in postmenopausal women who are at high risk for a fracture and for increasing bone mass in men with primary or hypogonadal osteoporosis who are at high risk for a fracture.

Approval Status: Approved by the U.S. FDA on November 26, 2002.

Chemical/Biological Nature: FORTEO is a recombinant human parathyroid hormone consisting of amino acids 1 to 34 [rhPTH(1-34)] of exactly the same N-terminal 34 amino acids of human parathyroid hormone. It is produced in E. coli and has a molecular weight of 4,117.8 daltons.

Administration: FORTEO is administered by subcutaneous ... Read more